FDA approved Atebrioz (zilurgisertib) to cut new heterotopic ossification volume in adults and children 12 and older with fibrodysplasia ossificans progressiva.
FDA approved Gazyva (obinutuzumab) to cut relapse risk in patients 2 years and older with frequently relapsing or steroid-dependent idiopathic nephrotic syndrome.
FDA approved AbbVie's Juvmo (tavapadon), a once-daily D1/D5 dopamine partial agonist pill, for Parkinson's disease in adults.
FDA approved Olumiant (baricitinib) for pediatric patients 12 years and older with severe alopecia areata, expanding the JAK inhibitor's US label.
FDA advisers will meet October 30 to review Sydnexis's NDA for atropine 0.01% eye drops to slow myopia progression in children 3 and older.
FDA approved belzutifan plus lenvatinib for adults with advanced clear cell renal cell carcinoma after PD-1 or PD-L1 inhibitor therapy.
FDA approves Onswik (insulin efsitora alfa-gobe), a once-weekly basal insulin, for glycemic control in adults with type 2 diabetes mellitus.
FDA approves lirafugratinib (Lyrfigtu) for previously treated, unresectable or metastatic cholangiocarcinoma harboring an FGFR2 fusion or rearrangement.
MHRA grants marketing authorisation to lurbinectedin with atezolizumab as maintenance in extensive-stage small cell lung cancer that has not progressed after induction.
Gossamer Bio files a new drug application with the FDA for seralutinib in pulmonary arterial hypertension; no filing acceptance or action date disclosed.
FDA approved a supplemental NDA for IntraBio's Aqneursa (levacetylleucine) for ataxia in adults and pediatric patients with ataxia-telangiectasia weighing at least 15 kg.
EMA's CHMP recommended 12 new medicines for approval and 11 indication extensions at its 14-17 September 2026 meeting, and upheld one refusal on re-examination.
FDA accepted LEO Pharma's dersimelagon NDA with Priority Review for EPP and XLP, with a target action date by end of February 2027.
FDA approved Eli Lilly's Inluriyo (imlunestrant) in combination with Verzenio (abemaciclib) for ER+/HER2-/ESR1-mutated advanced or metastatic breast cancer after prior endocrine therapy.
Xenon Pharmaceuticals has filed an NDA with the FDA for azetukalner in focal seizures and updated timelines for its psychiatric development programme.
MHRA issued a Class 4 medicines defect notification for Martindale clobazam oral suspension batches carrying an incorrect linear barcode; no recall is required.
FDA approved Ultragenyx's Fayuvi (rebisufligene etisparvovec-hopf) gene therapy for pediatric mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A) with preserved neurodevelopmental function.
FDA cites Korea Filler Experts for introducing unapproved and misbranded injectable botulinum toxin products, including Nabota 100UI, into interstate commerce.
FDA granted Fast Track designation to OncoC4's cesalatamig (AI-081), an investigational PD-1/VEGF bispecific antibody, for NSCLC after prior PD-(L)1 immunotherapy and platinum chemotherapy.
FDA approved Bayer's Kerendia (finerenone) for adults with chronic kidney disease associated with type 1 diabetes.
FDA approved Nuvation Bio's IBTROZI (taletrectinib) supplemental NDA updating the ROS1+ NSCLC label with mature duration-of-response data from TRUST-I and TRUST-II.
FDA approved Biofrontera's Ameluz (aminolevulinic acid HCl gel) with the BF-RhodoLED red-light lamp for superficial basal cell carcinoma, the first FDA-approved PDT for skin cancer.
FDA approved Curium's Bexlutry (lutetium Lu 177 dotatate) under 505(b)(2) for adults with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors.
FDA approves a label update for tarlatamab-dlle (Imdelltra) that reduces the post-dose monitoring period for the first two doses.
FDA approved Telix's Pixclara (floretyrosine F 18), the first FDA-approved FET-PET imaging agent for glioma, for adults and children 1 month and older.
FDA approved Scholar Rock's Isembyld (apitegromab-mstn) as the first muscle-targeted therapy for spinal muscular atrophy, as an add-on for patients 2+ on SMN2-targeted maintenance therapy.
FDA approved a supplemental NDA for Pharming's Joenja (leniolisib) extending the label to children ages 4-11 weighing at least 27 kg with activated PI3K delta syndrome.
FDA granted final approval to Handa Oncology's Omcazio (cabozantinib laurylsulfate) capsules under 505(b)(2), covering advanced RCC, HCC, and previously treated extra-pancreatic NETs.
FDA accepts Nanoscope Therapeutics' biologics license application for Mogenry in retinitis pigmentosa.
FDA issued a second Complete Response Letter for Seikagaku's condoliase (SI-6603) BLA in lumbar disc herniation, citing cGMP deficiencies at both drug-substance and drug-product facilities.
FDA granted accelerated approval to Bayer's HYRNUO (sevabertinib) as first-line therapy for adults with locally advanced or metastatic non-squamous HER2-mutated NSCLC.
FDA accepted Intellia Therapeutics' BLA for lonvoguran ziclumeran (lonvo-z), an in vivo CRISPR one-time treatment for hereditary angioedema, with Priority Review; PDUFA March 10, 2027.
FDA accepted Novartis's BLA for del-zota (delpacibart zotadirsen) under Priority Review, targeting the Accelerated Approval pathway for people with Duchenne muscular dystrophy amenable to exon 44 skipping. Filing rests on Phase 1/2 EXPLORE44 and EXPLORE44-OLE data.
FDA grants accelerated approval to camizestrant (Etcamah) plus a CDK4/6 inhibitor for HR-positive, HER2-negative advanced breast cancer with ESR1 mutations.
FDA approved Zanvastro (zilganersen) injection for the treatment of Alexander disease in pediatric and adult patients.
FDA directors warn that data from foreign clinical trial sites may be excluded from regulatory decisions if the agency cannot inspect to verify GCP compliance.
uniQure submitted a Biologics License Application to the FDA seeking accelerated approval of AMT-130 (ifezuntirgene inilparvovec), an investigational one-time gene therapy for Huntington's disease. A parallel Marketing Authorisation Application was submitted to the UK MHRA the same day. Filings are supported by three-year Phase I/II data showing slowed disease progression vs external control.
MHRA suspends the use, supply, and sale of Avacopan Vifor for new patients after concluding its pivotal trial data is unreliable.
FDA approved a labeling revision for ferric carboxymaltose injection (Injectafer) to add a boxed warning for symptomatic hypophosphatemia risk.
FDA issues draft guidance proposing a risk-based framework for establishing the credibility of AI models used to support regulatory decisions for drugs and biologics.
EMA launches a voluntary pilot for sponsors to submit data from non-animal testing methods and receive non-binding regulatory feedback.
FDA accepted Alvotech's BLA for AVT80, a proposed biosimilar to adalimumab.
FDA approves ropeginterferon alfa-2b-njft for the treatment of adults with essential thrombocythemia.
FDA issues M11 guidance on implementing harmonized digital clinical trial protocols to facilitate review.
FDA approved rusfertide for the treatment of polycythemia vera.
FDA approved tirzepatide (Mounjaro) to lower cardiovascular risk in adults with type 2 diabetes.
FDA approves ustekinumab for moderately to severely active ulcerative colitis in pediatric patients aged 2 years and older.
FDA accepted a New Drug Application for bimatoprost grenod for the treatment of open-angle glaucoma and ocular hypertension.
FDA approved Gilead's Bixlenvo (bictegravir 75 mg + lenacapavir 50 mg) as a once-daily single-tablet regimen for adults living with HIV who are virologically suppressed. Approval is based on the Phase 3 ARTISTRY-1 and ARTISTRY-2 trials.
FDA approved Priovant's Lisraya (brepocitinib) 30 mg once daily for adults with dermatomyositis, the first FDA-approved oral treatment and the first targeted therapy for the condition. Based on the Phase 3 VALOR trial.
FDA approved the XFG-adapted formulation of Novavax's Nuvaxovid COVID-19 vaccine for the 2026-2027 season, restricted to adults 65+ or ages 12-64 with a qualifying condition.
FDA approved supplemental BLAs for Moderna's updated 2026-2027 COVID-19 vaccine formulas.
FDA approved Pfizer and BioNTech's updated Comirnaty COVID-19 vaccine targeting the XFG variant.
FDA approves daraxonrasib for adults with metastatic pancreatic adenocarcinoma after prior therapy.
FDA approves expanded use of Roche's PATHWAY HER2 (4B5) and VENTANA HER2 Dual ISH tests to identify HER2-positive metastatic gastroesophageal adenocarcinoma patients eligible for treatment with ZIIHERA.
FDA granted Breakthrough Therapy Designation to GSK'227 (a B7-H3-targeted antibody-drug conjugate) for the treatment of relapsed or refractory osteosarcoma. Based on ARTEMIS-002 Phase 2 data.
FDA approves Tevimbra-based regimen for first-line treatment of HER2-positive gastroesophageal adenocarcinoma.
FDA approved dolutegravir oral suspension for treating HIV infection in babies from birth to four weeks old, based on data from the IMPAACT 2023 study.
FDA approves two zanidatamab-hrii-containing regimens for first-line treatment of adults with HER2-positive advanced gastroesophageal adenocarcinoma.
FDA extended the PDUFA for Capricor's deramiocel BLA from August 22 to November 22, 2026 after Capricor submitted a Major Amendment adding 24-month HOPE-3 open-label extension data and requesting a refined indication focused on upper-limb function.
FDA approved Johnson & Johnson's Imaavy (nipocalimab-aahu) for adults and pediatric patients aged 12+ with warm autoimmune hemolytic anemia (wAIHA) currently or previously treated with corticosteroids. First-ever approved therapy specifically for wAIHA.
FDA accepted United Therapeutics' NDA for ralinepag, an oral prostacyclin (IP) receptor agonist for pulmonary arterial hypertension. PDUFA target action date is June 24, 2027.
FDA grants Premarket Approval for Implantica's RefluxStop implant for the treatment of gastroesophageal reflux disease.
FDA approves RHA Redensity Eye hyaluronic acid filler for correction of moderate to severe tissue volume deficiencies in the infraorbital region.
FDA issued a complete response letter to Xspray Pharma's NDA for Dasynoc, a lower-dose bioequivalent formulation of dasatinib for CML and ALL, six days ahead of the Aug 25 PDUFA. The second consecutive CRL cites manufacturing observations at Xspray's contract manufacturer.
FDA accepted Teva's NDA and granted priority review for ecopipam, a first-in-class therapy for pediatric Tourette syndrome.
FDA grants accelerated approval to Genglycos (pariglasgene brecaparvovec-opnr) for patients aged 8 and older with glycogen storage disease type Ia.
FDA accepted Sedana Medical's NDA for isoflurane inhalation delivered via the Sedaconda ACD device for ICU sedation of mechanically ventilated adults.
FDA approves garetosmab-grts (Pasatru) to reduce new heterotopic ossification and disease flare-ups in adults with fibrodysplasia ossificans progressiva.
FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to Immatics' anzu-cel, a PRAME-directed TCR-T cell therapy, for unresectable or metastatic melanoma after prior anti-PD-1 therapy. RMAT confers all benefits of Breakthrough Therapy Designation plus early-interaction pathways specific to cell and gene therapies.
FDA approves an autoinjector version of lerodalcibep-liga and updates its indication for lowering cholesterol.
MHRA launches a pilot programme aiming to deliver first regulatory assessments for qualifying low-risk Phase 1 trials within 14 calendar days.
FDA approves Tauklarify (florquinitau F 18 injection), an F18-labeled tau PET imaging agent for use in Alzheimer's disease.
MHRA approves vimseltinib (romvimza) for adult patients with symptomatic tenosynovial giant cell tumour where surgery is not an option.
FDA granted accelerated approval to iberdomide in combination with daratumumab and hyaluronidase-fihj and dexamethasone for adults with relapsed or refractory multiple myeloma.
FDA accepted EIT Pharma's NDA for lonafarnib, a first-in-class oral farnesyltransferase inhibitor, for the treatment of chronic hepatitis D. The application is supported by the Phase 3 D-LIVR trial and carries Breakthrough Therapy, Fast Track, and Orphan Drug designations.
FDA issued a complete response letter to ITM for the NDA for 177Lu-edotreotide (ITM-11) in gastroenteropancreatic neuroendocrine tumors, citing CMC and third-party facility inspection items rather than clinical efficacy or safety.
MHRA authorises orforglipron (Foundayo) for weight management and type 2 diabetes, making the UK the first European country to approve the GLP-1 tablet.
FDA issues final ICH M11 guidance for harmonised digital clinical trial protocol content and exchange.
MHRA approves remibrutinib (Rhapsido) for adults with chronic spontaneous urticaria inadequately controlled by antihistamines.
FDA grants accelerated approval to oncolytic viral therapy Tudriqev in combination with nivolumab for advanced melanoma refractory to PD-1 blockade.
FDA approves Moderna's mRNA influenza vaccine mFLUSIVA for the prevention of seasonal influenza in adults.
FDA approved Takeda Pharmaceuticals's oveporexton (oveporexton) for narcolepsy type 1 on 2026-08-05.
FDA's Bioresearch Monitoring Program issued a Warning Letter to Maria E. De La Torre Silva, M.D., a clinical investigator in Homestead, Florida, citing two failure modes: - Double-enrollment: the site enrolled a subject in two overlapping drug trials at the same time, in direct violation of an explicit exclusion criterion prohibiting concurrent trial participation. - Fabricated dispensing records: EDC records marked six subjects as self-administering study drug nightly, but the sponsor's remote close-out visit found tamper-resistant seals on the IP kits still intact, meaning the drug was never dispensed or taken.
FDA approves Zimrixby (rituximab-cdxx), a biosimilar referencing Rituxan, for its oncology and autoimmune disease indications.
FDA approves lutetium Lu 177 vipivotide tetraxetan (Pluvicto) in combination with androgen receptor pathway inhibitor therapy for PSMA-positive metastatic prostate cancer.
MHRA grants marketing authorisation to aficamten for symptomatic obstructive hypertrophic cardiomyopathy.
FDA approves Garzulys, a biosimilar to NovoLog (insulin aspart), for glycemic control in diabetes.
FDA approved Lupin's generic sugammadex injection for the reversal of neuromuscular blockade induced by rocuronium bromide or vecuronium bromide in adults undergoing surgery, and Lupin launched the product commercially. Sugammadex is a selective relaxant-binding agent originally marketed as Merck's Bridion; multiple generics have entered the US market since 2023.
China's National Medical Products Administration (NMPA) accepted Kelun-Biotech's supplemental new drug application for sacituzumab tirumotecan (sac-TMT), a TROP-2-directed antibody-drug conjugate, as first-line treatment for adults with locally advanced or metastatic triple-negative breast cancer. sac-TMT was previously approved in China for pretreated advanced TNBC and non-squamous NSCLC. Gilead holds ex-China rights to sac-TMT through a 2024 partnership; the China 1L TNBC filing is Kelun's independent regulatory activity.
FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 10-3 on July 30, 2026 that the efficacy results from Replimune's IGNYTE study of RP1 (vusolimogene oderparepvec) in combination with nivolumab are evaluable and clinically meaningful for patients with advanced melanoma who have progressed on prior anti-PD-1 therapy. The favorable panel vote clears the review-level hurdle FDA had cited across the two prior CRL cycles (July 2025 and April 2026). The PDUFA target action date for the third-cycle resubmission is August 2, 2026 — three business days after the AdComm.
FDA licenses Ezplaz, the first freeze-dried plasma product for transfusion in adults when other plasma is unavailable.
FDA accepted PolyPid's New Drug Application for D-PLEX100 with Priority Review designation. D-PLEX100 is a locally delivered biodegradable polymer-lipid matrix that releases doxycycline directly at the surgical site over a controlled multi-week window, intended to prevent surgical site infections in abdominal colorectal surgery. Priority Review shortens the review clock to six months from the standard ten.
MHRA publishes a Public Assessment Report reviewing safety data on the risk of dementia associated with bladder anticholinergic medicines.
EMA's CHMP recommended 12 new medicines for approval at its 20-23 July 2026 meeting — 8 novel branded products (including three lipid-lowering approvals on the same day, Susvimo's port delivery implant, and the first oral IL-23R antagonist), one COVID-19 post-exposure antiviral, and three generics.
EMA's CHMP recommended 8 extensions of therapeutic indication at its 20-23 July 2026 meeting.
EMA's CHMP issued negative opinions for 3 applications at its 20-23 July 2026 meeting.
FDA approves MannKind's Furoscix ReadyFlow autoinjector for edema in adults with heart failure or chronic kidney disease.
FDA approves centanafadine (Simtriyo) for the treatment of attention-deficit hyperactivity disorder.
FDA granted Fast Track designation to Lundbeck's Lu AH69593, an investigational oral orexin 2 receptor agonist, for the treatment of narcolepsy. Orexin 2 signalling drives wakefulness; Lundbeck is one of several sponsors (Takeda, Centessa, Alkermes) pursuing OX2R agonism as a mechanism-targeted approach to narcolepsy type 1 (orexin-deficient) and adjacent hypersomnias.
FDA accepted BridgeBio's New Drug Application for encaleret, an oral calcium-sensing receptor antagonist, in autosomal dominant hypocalcemia type 1 (ADH1), with a PDUFA target action date of May 8, 2027. FDA is not currently planning to hold an advisory committee. If approved, encaleret would become the first therapy specifically indicated for ADH1, a rare genetic form of hypoparathyroidism driven by gain-of-function CASR mutations.
FDA accepted Revolution Medicines' NDA for daraxonrasib in previously treated metastatic pancreatic cancer.
FDA approves zidesamtinib for adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer who have received prior systemic therapy.
MHRA updates product information for domperidone to contraindicate use in patients with confirmed or suspected phaeochromocytoma due to risk of severe hypertension.
MHRA approves new versions of Clenil Modulite inhalers using a next-generation, lower-carbon propellant for asthma maintenance.
FDA approved Outlook Therapeutics's LYTENAVA (bevacizumab-vikg) for neovascular (wet) age-related macular degeneration on 2026-07-21.
FDA approves expanded use of Ryaltris nasal spray for children aged 6 to less than 12 years.
FDA accepted Dyne Therapeutics's Biologics License Application for z-rostudirsen (DYNE-251) with Priority Review, seeking Accelerated Approval for adults and children with Duchenne muscular dystrophy amenable to exon 51 skipping. PDUFA target action date is January 21, 2027.
FDA accepted Camurus's NDA resubmission for CAM2029 (Oclaiz, octreotide extended-release subcutaneous) in acromegaly on July 17, 2026, restarting the review clock after the June 10 CRL. Class 2 review with a PDUFA target action date of December 18, 2026.
FDA grants traditional approval to Novartis's Fabhalta (iptacopan) to slow kidney function decline in adults with primary immunoglobulin A nephropathy.
FDA approves Lipfendra (enlicitide) as the first oral PCSK9 inhibitor for adults with high cholesterol or heterozygous familial hypercholesterolemia.
FDA accepted Apnimed's NDA for AD109, an oral treatment for adults with obstructive sleep apnea.
FDA approved Revtorpyk (gedatolisib) for HR+/HER2-, PIK3CA wild-type locally advanced or metastatic breast cancer.
FDA granted traditional approval to selpercatinib for locally advanced or metastatic RET fusion-positive solid tumors in adults and pediatric patients aged two and older.
FDA cleared a subcutaneous starting dose regimen for Eisai’s Leqembi, enabling home administration for Alzheimer’s patients.
FDA accepted BioMarin's supplemental NDA for VOXZOGO (vosoritide) seeking full approval — converting from the 2021 accelerated approval — in children with achondroplasia. PDUFA target action date is February 28, 2027.
FDA issued a Complete Response Letter for rivoceranib plus camrelizumab as first-line treatment for unresectable or metastatic hepatocellular carcinoma.
FDA approved isatuximab-irfc for subcutaneous injection in combination with various regimens for multiple myeloma indications.
NMPA's Center for Drug Evaluation admitted Leads Biolabs' BLA for opamtistomig (LBL-024, PD-L1/4-1BB bispecific antibody) to priority review as monotherapy for advanced EP-NEC in patients whose disease progressed on 2+ prior systemic therapy lines.
FDA approved pembrolizumab or pembrolizumab with berahyaluronidase alfa-pmph, each combined with enfortumab vedotin-ejfv, for neoadjuvant and adjuvant treatment of muscle-invasive bladder cancer.
EMA's PRAC issued new safety recommendations for desogestrel- and etonogestrel-containing contraceptives regarding meningioma risk and strengthened warnings for Litfulo's JAK inhibitor risks.
GSK's licensor Hansoh Pharma reported positive Phase III ARTEMIS-008 topline: risvutatug rezetecan (Ris-Rez) improved overall survival vs topotecan in advanced/relapsed SCLC (China patient population). Ris-Rez, a B7-H3-targeted ADC licensed to GSK ex-Greater-China, holds two FDA Breakthrough Therapy Designations (r/r ES-SCLC + r/r osteosarcoma) and an EMA PRIME designation for r/r ES-SCLC.
FDA schedules Cellular, Tissue, and Gene Therapies Advisory Committee meeting to discuss Replimune's BLA 125827 for vusolimogene oderparepvec.
MHRA approved nerandomilast (Jascayd) for idiopathic pulmonary fibrosis and progressive pulmonary fibrosis in adults.
FDA approved wilate for routine prophylaxis in children under 6 with von Willebrand disease.
FDA accepted Arcutis's supplemental NDA for ZORYVE (roflumilast) cream 0.05% to expand the topical mild-to-moderate atopic dermatitis indication to infants aged 3 to 24 months. PDUFA target action date is February 23, 2027.
MHRA authorised UCB's Kygevvi (doxecitine + doxribtimine) as the first treatment for paediatric and adult patients with thymidine kinase 2 deficiency (TK2d) with age of symptom onset on or before 12 years. TK2d is an ultra-rare mitochondrial disease that impairs the maintenance of mitochondrial DNA in muscle and produces progressive skeletal muscle weakness — historically with no approved disease-modifying therapy.
EMA's Committee for Medicinal Products for Human Use (CHMP) has initiated a phased review of Revolution Medicines' daraxonrasib for the treatment of metastatic pancreatic cancer in patients who have received prior treatment. Phased review is EMA's accelerated-assessment path in which data are evaluated as they become available, compressing overall timeline. The decision was made on the basis of a Phase 3 study published in the New England Journal of Medicine comparing daraxonrasib to chemotherapy, in a patient population with a life expectancy of about 6 months after prior-line progression.
FDA granted accelerated approval to Trutakna (atacicept-vymj) for adult patients with primary IgA nephropathy.
FDA accepted Organogenesis Holdings's Biologics License Application for ReNu — a cryopreserved amniotic suspension allograft — for the management of pain in symptomatic knee osteoarthritis. The PDUFA target action date is set for April 24, 2027. If approved, ReNu would be the first FDA-approved non-surgical biologic therapy for knee OA, an indication with ~31 million U.S. patients and no regenerative-therapy incumbent.
FDA accepted Pharvaris's New Drug Application for deucrictibant immediate-release (20 mg) for the on-demand treatment of hereditary angioedema (HAE) attacks in patients 12 years and older. The PDUFA target action date is set for April 23, 2027. Deucrictibant is a novel, potent, orally bioavailable small molecule bradykinin B2 receptor antagonist — a mechanism-of-action that would be unique among oral on-demand HAE treatments if approved.
MHRA authorised Incyte's Zynyz (retifanlimab) for the treatment of adults with metastatic or recurrent locally advanced Merkel cell carcinoma. Zynyz is a humanised anti-PD-1 monoclonal antibody previously FDA-approved for this indication in March 2023; the UK authorisation adds the second major-market approval for a treatment class that has substantially reshaped outcomes in an aggressive skin cancer historically treated with chemotherapy.
FDA issued a Complete Response Letter for Ascelia Pharma's Orviglance (manganese chloride tetrahydrate) NDA for contrast-enhanced liver MRI in patients with severe kidney impairment, citing deficiencies in clinical data (image reading process) and product documentation.
MHRA granted conditional marketing authorisation to Novo Nordisk's Wegovy (semaglutide 2.4 mg weekly injection) for adults with metabolic dysfunction-associated steatohepatitis (MASH) and moderate-to-advanced liver fibrosis. This is the first UK approval of a GLP-1 receptor agonist for MASH — a chronic liver disease historically without a marketed pharmacological treatment — and extends Wegovy beyond its established weight-management and cardiovascular-risk-reduction indications.
FDA issued a supplemental approval expanding Casgevy (exagamglogene autotemcel) to patients aged 2 years and older with sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent β-thalassemia. Casgevy was previously approved for ages 12+, and this becomes the first gene therapy approved for patients as young as 2 with SCD.
MHRA authorised Inqlexzo, an intravesical gemcitabine-releasing device, for adults with high-risk non-muscle-invasive bladder cancer (HR-NMIBC) that has not responded to standard BCG therapy. The device delivers gemcitabine continuously to the bladder over ~3 weeks and is positioned for patients ineligible for — or declining — radical cystectomy.
FDA grants interchangeability status to Celltrion's Truxima (rituximab), the first biosimilar to achieve this designation.
FDA approved Orca Bio's Tregzi for adult patients undergoing matched-donor allogeneic hematopoietic stem cell transplantation with a myeloablative preparative regimen — the first precision-engineered allogeneic regulatory T cell therapy authorized in the U.S. Approval covers hematopoietic and immunologic reconstitution and improvement of chronic graft-versus-host-disease-free survival.
FDA issued a Complete Response Letter to Unicycive Therapeutics for the resubmitted NDA of oxylanthanum carbonate (OLC) in hyperphosphatemia. FDA cited the same third-party manufacturing deficiencies flagged in the June 2025 CRL and confirmed no clinical efficacy or safety concerns were raised; the agency's inspection of the third-party vendor had not been conducted as part of this review cycle.
FDA's CDER Small Business and Industry Assistance (SBIA) program has published the materials from its April 15, 2026 webinar on the Expanded Access pathway for investigational drugs. The session covers the three EA categories (individual patient, intermediate-size population, treatment INDs and protocols) along with submission requirements, sponsor responsibilities, and common misconceptions.
FDA extended by three months the review period for Praxis Precision Medicines's relutrigine NDA, updating the PDUFA target action date from September 27, 2026 to December 27, 2026. Praxis submitted additional sensitivity analyses of existing clinical data, which FDA classified as a 'major amendment'. FDA raised no new safety or manufacturing concerns and requested no additional clinical studies.
FDA approved Merz Therapeutics' Xeomin (incobotulinumtoxinA) for the treatment of upper-limb spasticity in pediatric patients aged 2 and older with cerebral palsy. Xeomin was previously approved for adult upper-limb spasticity in 2010; this label expansion extends the indication into the largest population for which upper-limb spasticity intervention is prescribed.
FDA approved Arcutis's Zoryve (roflumilast) cream 0.3% for the topical treatment of plaque psoriasis in children aged 2 and older, expanding the label from its prior adult and adolescent indications.
FDA's Cellular, Tissue, and Gene Therapies Advisory Committee will meet July 29, 2026 to review Capricor's BLA 125842 for deramiocel, an allogeneic cardiosphere-derived cell therapy for Duchenne muscular dystrophy. PDUFA target action date is August 22, 2026.
FDA approved PharmaEssentia's BESREMi Pen (ropeginterferon alfa-2b-njft) for polycythemia vera treatment.
EMA's CHMP issued positive opinions for therapeutic-indication extensions on 12 already-authorised medicines at its 22-25 June 2026 meeting, including expansions for Rinvoq, Stelara, Tecvayli, Datroway, Leqvio, Jaypirca, Opzelura, Imvanex, Menquadfi, Rezolsta, and Symtuza.
EMA's CHMP recommended six new medicines for approval, including Aujemflu for influenza and Hopledo for Parkinson's disease, and extended indications for 11 existing products.
EMA's CHMP issued negative opinions for three applications at its 22-25 June 2026 meeting: Tacquell (autologous TIL therapy for advanced melanoma), Yartemlea (Omeros narsoplimab for HSCT-TMA), and Xervyteg (MaaT Pharma fecal microbiota for acute graft-versus-host disease).
FDA issued a Complete Response Letter for Sobi's NASP (nanoencapsulated sirolimus + pegadricase) BLA in adults with uncontrolled gout, citing deficiencies at contract manufacturing facilities and additional data on the biologic manufacturing control strategy — no clinical efficacy or safety concerns were raised.
FDA approved Viridian's Lumvoa (veligrotug-vvze) for the treatment of thyroid eye disease in adults — the first treatment with a label covering both active and chronic forms of the disease.
FDA accepted Replimune's resubmission of the RP1 (vusolimogene oderparepvec) Biologics License Application for advanced melanoma as a Class 1 complete response with a PDUFA target action date of August 2, 2026. FDA notified Replimune to expect an advisory committee meeting in late July. This reverses course after the initial April 2026 review cycle that culminated in a CRL.
FDA approved Skyrizi (risankizumab-rzaa) for pediatric patients aged 6 and older with moderate-to-severe plaque psoriasis or active psoriatic arthritis, expanding its existing adult indications into the pediatric age group.
FDA approved palbociclib with trastuzumab, with or without pertuzumab, and endocrine therapy for maintenance treatment of HR-positive, HER2-positive metastatic breast cancer.
FDA approved sacituzumab govitecan-hziy as monotherapy and in combination with pembrolizumab for first-line treatment of unresectable locally advanced or metastatic triple-negative breast cancer.
FDA approved Tryngolza (olezarsen) to reduce triglycerides and acute pancreatitis risk in adults with severe hypertriglyceridemia.
FDA cites Novo Nordisk for misleading promotional claims about Wegovy, Ozempic, and Victoza in a direct-to-consumer video.
FDA issued a Complete Response Letter to Achieve Life Sciences for the cytisinicline NDA. The sponsor plans a Q4 2026 resubmission.
FDA outlines framework for using digital health technologies in clinical trials, including decentralized trials and remote data acquisition.
FDA seeks public feedback on a proposed Expedited IND pilot program to streamline first-in-human trials and reduce clinical holds.
FDA issued guidance on the design, analysis, and regulatory submission of clinical trials conducted under master protocols.
FDA released a Phase 1 IND Navigator tool to assist sponsors in preparing and submitting Investigational New Drug applications.
FDA issued guidance on using quantitative systems pharmacology to determine MABEL dose in first-in-human trials.
FDA issued a revised draft guidance clarifying the substantial evidence of effectiveness standard for drug and biologic approvals.
Ipsen voluntarily withdrew Tazverik's three FDA accelerated-approval indications after SYMPHONY-1 trial showed an increased rate of second primary malignancies. Effective via Federal Register 2026-06-22.
FDA converted Tecelra's 2024 accelerated approval to full (traditional) approval and expanded the indication to include patients as young as 12 years — the first engineered T-cell therapy for a solid tumour approved for a paediatric population. Full approval was granted on the basis of the SPEARHEAD-1 confirmatory Phase 2 trial (n=137) which produced an overall response rate of 43.8% (complete responses 3.6%) in HLA-A*02-eligible, MAGE-A4-expressing advanced synovial sarcoma. Sponsor is US WorldMeds, which acquired Tecelra's U.S. commercial rights from Adaptimmune.
FDA approved Merck's Capvaxive (21-valent pneumococcal conjugate vaccine) for children and adolescents aged 2-17 at increased risk for pneumococcal disease. The approval expands Capvaxive from its original adult indication into the pediatric high-risk population.
FDA cleared Norwich Pharmaceuticals’ generic baloxavir marboxil tablets for acute uncomplicated influenza treatment and prophylaxis in patients 5 years and older.
FDA approved Utebzi (tebipenem pivoxil) tablets for complicated urinary tract infections, including pyelonephritis, in adults with limited oral treatment options.
FDA acknowledged receipt of Outlook Therapeutics's resubmitted Biologics License Application for ONS-5010/LYTENAVA (bevacizumab-vikg) as a treatment for neovascular (wet) age-related macular degeneration. The resubmission is a Class 1 review with a PDUFA target action date of July 29, 2026 — approximately three weeks out. If approved, LYTENAVA would be the first and only ophthalmic formulation of bevacizumab with FDA-approved labelling and standardised manufacturing, replacing the off-label use of oncology-formulation Avastin that currently dominates the wet-AMD anti-VEGF injection market.
FDA approved Amphastar's Rextovy, an over-the-counter naloxone nasal spray, for emergency treatment of opioid overdose.
FDA approved Bayer's Ambelvist (gadoquatrane) for use with contrast-enhanced MRI to detect and visualize lesions with abnormal vascularity in the central nervous system and non-CNS body regions. Ambelvist is dosed at 0.01 mmol/kg — the lowest-dose macrocyclic gadolinium-based contrast agent ever approved in the United States.
FDA accepted Gilead Sciences's supplemental New Drug Application for Yeztugo (lenacapavir) 300 mg tablet as a once-weekly oral formulation for HIV pre-exposure prophylaxis (PrEP). PDUFA target action date is February 2, 2027. If approved, once-weekly oral Yeztugo would be the first long-acting oral PrEP option — filling the middle ground between the current daily-oral standard-of-care (F/TDF, F/TAF) and Gilead's own twice-yearly subcutaneous lenacapavir (Yeztugo injection, approved 2025). The submission is supported by the established clinical profile of lenacapavir in PrEP from the PURPOSE 1 and PURPOSE 2 Phase 3 trials.
FDA approved belzutifan in combination with pembrolizumab for adjuvant treatment of renal cell carcinoma with a clear cell component at intermediate-high or high risk of recurrence.
FDA approved capivasertib with abiraterone and prednisone for metastatic PTEN-deficient prostate cancer, alongside a companion diagnostic.
FDA approved Keytruda (pembrolizumab, IV) and Keytruda Qlex (pembrolizumab + berahyaluronidase alfa-pmph, subcutaneous), each in combination with Welireg (belzutifan), for adjuvant treatment of adults with clear cell renal cell carcinoma at intermediate-high or high risk of recurrence following nephrectomy. This is the first approved combination of a PD-1 inhibitor and a HIF-2α inhibitor.
MHRA authorised a 1 mg nasal spray formulation of EURneffy for emergency treatment of anaphylaxis in children aged 4+ weighing 15-30 kg.
EMA’s PRAC recommends maintaining precautionary measures for valproate use in men pending further study results, citing inconsistent evidence on neurodevelopmental risks.
FDA cleared teplizumab (Tzield) for delaying the onset of stage 3 type 1 diabetes in pediatric patients.
MHRA approved semaglutide tablets for weight loss and management in adults with obesity or overweight with comorbidities.
FDA issued a complete response letter for Camurus’ NDA for CAM2029 (Oclaiz) in acromegaly.
FDA approved Tofidence for cytokine release syndrome and pediatric COVID-19 based on Organon's supplemental biologics license application.
FDA approved a maintenance dosing regimen of Ebglyss (lebrikizumab-lbkz) every eight weeks for moderate-to-severe atopic dermatitis.
GSK announced a $10.6B all-cash agreement to acquire Nuvalent, gaining ROS1 inhibitor zidesamtinib (PDUFA 2026-09-18) and ALK inhibitor neladalkib (PDUFA 2026-11-27). Both NSCLC candidates carry FDA Breakthrough Therapy and Orphan Drug designations.
MHRA authorised nipocalimab for adults and adolescents aged 12+ with generalised myasthenia gravis based on evidence from clinical trials.
FDA issued a Warning Letter to Jose M. Carpio, M.D. for failing to adhere to protocol requirements in clinical investigations of an unnamed investigational drug.
FDA mandates updates to Boxed Warnings for Xeljanz, Olumiant, and Rinvoq to include risks of serious heart-related events, cancer, blood clots, and death.
FDA approved Pfizer's Hympavzi for additional hemophilia A and B patient populations based on significant medical need.
FDA warns of rare liver decompensation cases in Hepatitis C patients with moderate to severe liver impairment treated with Mavyret, Zepatier, or Vosevi.
MHRA reviews avacopan's benefit-risk profile following concerns about pivotal trial data integrity.
FDA approved Lupin's Ranluspec (ranibizumab-hkdz) as an interchangeable biosimilar to Lucentis for the treatment of neovascular (wet) age-related macular degeneration, macular oedema, and other approved Lucentis indications.
MHRA granted marketing authorisation to Seqirus UK for an adjuvanted trivalent influenza vaccine for adults aged 50 and over.
Teva UK issued a Class 4 Medicines Defect Notification for Ponlimsi (denosumab) 60mg pre-filled syringes due to a carton labelling error stating 'For application to the skin' instead of subcutaneous use.
CDER encourages sponsors to propose nonclinical studies using New Approach Methodologies to reduce animal testing in drug development.
FDA posts condition-specific meeting reports and resources from externally-led PFDD meetings and patient listening sessions.
FDA launches C3TI Compass, a centralized repository of guidance documents, case studies, and resources supporting innovative clinical trial design and conduct.
EMA’s Emergency Task Force engages with African regulators to advance clinical trials for Ebola treatments and vaccines targeting the Bundibugyo virus.
FDA accepts Letter of Intent for an AI-driven digital liver model to predict drug-induced liver injury under the ISTAND qualification program.
FDA finalizes guidance for sponsors developing antibacterial drugs to treat pulmonary tuberculosis.
FDA cites real-world evidence in regulatory decisions for Acetadote, Onapgo, Isturisa, and Gamifant dosing and indication changes.
FDA updates labeling for multiple drugs based on real-world evidence from postmarketing studies assessing pregnancy outcomes, opioid risks, and long-term safety.
MHRA authorised resmetirom (Rezdiffra) for adult patients with metabolic dysfunction-associated steatohepatitis (MASH) and moderate to advanced liver fibrosis.
FDA approved Cavhanza (nilotinib) orally disintegrating tablets from Cycle Pharmaceuticals for Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML). The ODT formulation is designed to be taken without regard to meals or acid-reducing agents (PPIs and H2 antagonists), removing the food-effect and drug-interaction restrictions that constrain the reference-listed nilotinib product (Tasigna).
FDA released draft guidance to streamline regulatory submissions for cell and gene therapies by leveraging existing scientific and platform knowledge.
FDA issues final guidance on general principles for model-informed drug development, harmonizing assessment frameworks and regulatory interactions.
FDA approved Cypsedo (cipepofol) injection from Haisco Pharmaceutical Group for general anesthesia. Cipepofol is a short-acting GABA-A positive allosteric modulator ~4-6× more potent than propofol, and Cypsedo is the first China-originated innovative intravenous anesthetic to receive FDA marketing authorization.
FDA's Oncologic Drugs Advisory Committee will discuss AstraZeneca's camizestrant NDA and Truqap sNDA for breast and prostate cancer indications on April 30, 2026.
FDA approved Xocova (ensitrelvir) for post-exposure prophylaxis of COVID-19, marking the first oral option for this indication.
FDA approved Zaynich (cefepime and zidebactam) for adults with complicated urinary tract infections.
FDA approved inhaled insulin Afrezza for children and adolescents aged 6+ with diabetes.
EMA’s Emergency Task Force recommends updating COVID-19 vaccines to target the XFG variant for the 2026/2027 vaccination campaign.
FDA proposes streamlined nonclinical safety studies for oncology biologics and conjugated products, aiming to reduce unnecessary animal testing.
MHRA grants marketing authorisation for rilzabrutinib to treat adults with immune thrombocytopenia when prior therapies are insufficient.
FDA extends the review period for Beren Therapeutics' NDA for adrabetadex in infantile-onset Niemann-Pick disease type C.
FDA approved AstraZeneca's Imfinzi in combination with BCG induction and maintenance therapy for BCG-naïve, high-risk non-muscle-invasive bladder cancer.
FDA approved a supplemental BLA for Tremfya to include evidence supporting its efficacy in preventing further joint damage.
FDA removed the Boxed Warning about leg and foot amputation risk from canagliflozin (Invokana) labeling based on new clinical trial data showing enhanced heart and kidney benefits.
FDA required revisions to the Boxed Warning for Xeljanz, Olumiant, and Rinvoq to include risks of serious heart-related events, cancer, blood clots, and death based on a safety clinical trial.
FDA removed the REMS program for clozapine, no longer requiring ANC blood test reporting but maintaining monitoring recommendations.
FDA approved Linzess (linaclotide) for pediatric patients aged 2+ with functional constipation.
FDA approved pivekimab sunirine-pvzy for adults with blastic plasmacytoid dendritic cell neoplasm based on CADENZA trial results.
FDA requested removal of the contraindication against statin use during pregnancy, allowing individualized benefit-risk decisions for high-risk patients.
FDA alerts public to preliminary findings of increased heart-related events and cancer risk with Xeljanz compared to TNF inhibitors.
FDA requires updated Boxed Warnings and prescribing information for all benzodiazepines to address risks of abuse, addiction, dependence, and withdrawal.
FDA cautions against using unapproved compounded versions of GLP-1 drugs like semaglutide and tirzepatide due to safety and quality concerns.
FDA updates guidance documents on rare disease drug development, covering topics from natural history studies to accelerated approval pathways.
FDA removed REMS requirements for embryofetal toxicity risk from ambrisentan, macitentan-containing products, and aprocitentan, citing sufficient risk communication through labeling.
FDA approved Differin Epiduo Acne Gel (adapalene/benzoyl peroxide) for over-the-counter treatment of acne.
FDA cleared datopotamab deruxtecan-dlnk for unresectable or metastatic triple-negative breast cancer.
EMA's CHMP recommended eight medicines for approval, including Jascayd, Boey, Etcamah, and Vijoice, at its May 2026 meeting.
FDA approved Hepcludex (bulevirtide-gmod) injection for chronic hepatitis delta virus infection in adults without cirrhosis or with compensated cirrhosis.
EMA recommended marketing authorisation for Jascayd (nerandomilast) to treat idiopathic pulmonary fibrosis and other fibrotic lung diseases.
FDA issued a Warning Letter to CSL Behring for advertising and promotional labeling violations.
FDA accepted Bayer's NDA for Factor XIa inhibitor asundexian for prevention of thromboembolic events.
FDA issued a Warning Letter to clinical investigator Naseem A. Jaffrani for violations identified during an inspection.
FDA issued a Warning Letter to Sourav K. Mishra, M.D. regarding deficiencies in bioavailability-bioequivalence studies.
FDA approved AstraZeneca's Baxfendy (baxdrostat) as the first aldosterone synthase inhibitor for hypertension in combination with other antihypertensive medications.
FDA approved Immgolis (golimumab-sldi) as an interchangeable biosimilar to Simponi for Accord BioPharma.
FDA approved Immgolis Intri (golimumab-sldi) as an interchangeable biosimilar to Simponi Aria (golimumab).
FDA approved Trimbow (beclomethasone/formoterol/glycopyrrolate) inhaler for maintenance treatment of asthma.
MHRA approved beremagene geperpavec (Vyjuvek) for the treatment of dystrophic epidermolysis bullosa.
FDA approved Enhertu for two new indications in HER2-positive breast cancer patients in neoadjuvant and adjuvant settings.
FDA approved Genentech's Tecentriq for adjuvant treatment of muscle-invasive bladder cancer using ctDNA-guided therapy.
FDA approved a dosing update for Crysvita (burosumab-twza) in adults with X-linked hypophosphatemia.
FDA approved AstraZeneca's Fasenra for adult and pediatric patients aged 12+ with hypereosinophilic syndrome without an identifiable non-hematologic cause.
FDA granted accelerated approval to Beqalzi (sonrotoclax) for relapsed or refractory mantle cell lymphoma.
FDA approved Inqovi (decitabine/cedazuridine) plus venetoclax for acute myeloid leukemia patients ineligible for intensive induction chemotherapy.
FDA approved Bizengri (zenocutuzumab-zbco) for NRG1 fusion-positive cholangiocarcinoma under the National Priority Voucher Pilot Program.
FDA approved Ocrevus (ocrelizumab) IV infusion for relapsing-remitting multiple sclerosis in pediatric patients aged 10 and older.
FDA issued final guidance on methodologies for postapproval pregnancy safety studies of drugs and biologics.
FDA approved Vyvgart and Vyvgart Hytrulo for all adult patients with generalized myasthenia gravis.
EMA issued recommendations for egg-derived and live-attenuated influenza vaccine strains for the 2026/2027 season.
FDA cleared Ennumo (pegfilgrastim-pccg) as a biosimilar to Neulasta for reducing infection incidence in chemotherapy patients.
FDA awarded a Commissioner’s National Priority Voucher to Partner Therapeutics for Zenocutuzumab in NRG1 fusion-positive cholangiocarcinoma.
FDA approved Jakafi XR (ruxolitinib) extended-release tablets for myelofibrosis, polycythemia vera, and graft-versus-host disease in adults.
FDA approved Veppanu (vepdegestrant) for the treatment of ESR1m, ER+/HER2- advanced breast cancer.
FDA approved an expanded indication for Asceniv to include pediatric immune-compromised patients aged two years and older.
FDA approved Auvelity (dextromethorphan/bupropion) for agitation associated with dementia due to Alzheimer's disease.
FDA proposes to withdraw approval of ChemoCentryx's TAVNEOS (avacopan) due to lack of substantial evidence and material misstatements in the application.
FDA approved Langlara (insulin glargine-aldy) as an interchangeable biosimilar to Lantus for diabetes treatment.
FDA announced two major steps to implement real-time clinical trials as part of a broader initiative to modernize trial conduct.
FDA approved a supplemental New Drug Application for Caplyta (lumateperone) based on long-term data supporting reduced relapse risk in schizophrenia.
FDA approved AstraZeneca's Saphnelo Pen autoinjector for once-weekly subcutaneous self-administration in adult SLE patients.
EMA's CHMP issued positive opinions for Sanofi's tolebrutinib, Novartis' onasemnogene abeparvovec, and Arrowhead's plozasiran across three indications.
EMA recommended granting marketing authorisation for Redemplo (plozasiran) to treat adults with familial chylomicronaemia syndrome.
FDA approved Otarmeni, a dual AAV vector-based gene therapy, for genetic hearing loss under the National Priority Voucher Program.
FDA approved Dupixent (dupilumab) for children aged two to 11 years with chronic spontaneous urticaria who remain symptomatic despite antihistamine treatment.
FDA approved Tzield (teplizumab-mzwv) for delaying stage 3 type 1 diabetes onset in children as young as one year old.
FDA approved Idvynso, a two-drug single-tablet regimen of doravirine and islatravir, for HIV-1 treatment in adults.
FDA approved an expanded indication for Caldolor (ibuprofen) injection based on Cumberland Pharmaceuticals' application.
FDA invites sponsors of approved testosterone replacement therapies to discuss potential new indications for low libido in men with idiopathic hypogonadism.
FDA extended the review period for Savara's Molgramostim Inhalation Solution BLA in autoimmune pulmonary alveolar proteinosis.
FDA approved ustekinumab for patients aged two years and older with moderately to severely active Crohn’s disease.
FDA released draft guidance on safety standards for genome editing technologies in human gene therapy product development.
FDA approved Filspari (sparsentan) to reduce proteinuria in adult and pediatric patients with focal segmental glomerulosclerosis.
FDA approved an updated label for Vabysmo (faricimab-svoa) to extend treatment duration for macular edema following retinal vein occlusion beyond six months.
FDA approved extended dosing intervals up to 5 months for Eylea HD (aflibercept) in wet age-related macular degeneration and diabetic macular edema.
FDA granted full approval to Kite's CAR T-cell therapy Tecartus for adult patients with relapsed or refractory mantle cell lymphoma.
FDA approved expanded use of Alyftrek and Trikafta for cystic fibrosis treatment, increasing availability to ~95% of patients.
FDA approved orforglipron as the first new molecular entity under the Commissioner's National Priority Voucher pilot program.
FDA cleared Ponlimsi (denosumab-adet) as a biosimilar to Prolia for osteoporosis treatment.
FDA approved a high-dose regimen of Spinraza (nusinersen) for spinal muscular atrophy.
FDA granted accelerated approval to Rocket Pharmaceuticals' Kresladi for pediatric patients with severe leukocyte adhesion deficiency-I.
FDA approved removal of the age requirement from the Neffy (epinephrine nasal spray) label, expanding its use across all age groups.
FDA approved Awiqli (insulin icodec-abae) as the first once-weekly basal insulin treatment for adults with Type 2 diabetes.
FDA granted accelerated approval to Denali Therapeutics' Avlayah (tividenofusp alfa-eknm) for the treatment of Hunter syndrome (MPS II).
FDA approved Lifyorli (relacorilant) in combination with nab-paclitaxel for platinum-resistant ovarian cancer.
FDA lifted its previously issued Complete Response Letter for Capricor Therapeutics' deramiocel BLA and accepted the resubmission as a Class 2 with a PDUFA target action date of August 22, 2026.
FDA accepted Savara's BLA for Molbreevi in autoimmune pulmonary alveolar proteinosis for review.
FDA issued a Complete Response Letter to Disc Medicine for bitopertin in erythropoietic protoporphyria.
FDA issued a Complete Response Letter to Aquestive Therapeutics for Anaphylm, citing deficiencies in the NDA.