CHMP recommends approval for tolebrutinib, onasemnogene abeparvovec, plozasiran
- Sponsor
- Sanofi Winthrop Industrie, Novartis Europharm Limited, Arrowhead Pharmaceuticals Ireland Limited
- Drug
- tolebrutinib, onasemnogene abeparvovec, plozasiran
- Indication
- non-relapsing secondary progressive MS, 5q spinal muscular atrophy, familial hypercholesterolemia
EMA's CHMP issued positive opinions for Sanofi's tolebrutinib, Novartis' onasemnogene abeparvovec, and Arrowhead's plozasiran across three indications.
What this means
Tolebrutinib is an oral BTK inhibitor that may slow nerve damage in progressive MS, while onasemnogene abeparvovec is a gene therapy replacing the faulty SMN1 gene in spinal muscular atrophy, and plozasiran is an RNA interference drug that reduces liver production of a cholesterol-raising protein. The EMA's expert panel recommended approving all three drugs for their respective conditions, moving them closer to final authorization in the EU. These decisions could provide new treatment options for progressive MS patients without relapses, babies with a severe genetic muscle-wasting disease, and people with inherited high cholesterol resistant to standard therapies.
From the source
Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 20-23 April 2026
24 April 2026
Five new medicines recommended for approval; another nine medicines recommended for extension of their therapeutic indications
NewsHumanCOVID-19Medicines
Update as of 18 May 2026
'CHMP statistics' graphic - updated figures for total negative opinions on new medicines and for positive opinions on extensions of therapeutic indications in 2026.
Five new medicines recommended for approval
EMA’s human medicines committee (CHMP) recommended five medicines for approval at its April 2026 meeting.
The committee recommended granting a marketing authorisation for Cenrifki (tolebrutinib), for the treatment of non-relapsing secondary progressive multiple sclerosis, a disease of the brain and spinal cord in which inflammation destroys the protective covering around nerves and the nerves themselves.
The CHMP recommended granting a marketing authorisation for the gene therapy medicine Itvisma (onasemnogene abeparvovec), for the treatment of spinal muscular atrophy, a rare, serious inherited disease that causes weakness and wasting of the muscles.
The CHMP adopted a positive opinion for Redemplo (plozasiran) to treat adults with familial chylomicronaemia syndrome, a rare inherited disease that prevents the body from breaking down lipids (fats). This medicine provides a new treatment option for patients with a high unmet medical need. See more details in the news announcement in the grid below.
The committee recommended granting a marketing authorisation for Rexatilux (ranibizumab), a biosimilar medicine for the treatment of several eye diseases causing vision impairment.
A generic medicine, Palbociclib Viatris (palbociclib), received a positive opinion for the treatment of breast cancer.
Recommendations on extensions of therapeutic indication for nine medicines
The committee recommended extensions of indication for nine medicines that are already authorised in the European Union (EU): Agamree, Aquipta, Crysvita, Comirnaty, Inaqovi, Opdivo, Privigen, Skyrizi and Venclyxto.
Withdrawal of applications
An application for an initial marketing authorisation was withdrawn. Viokat (diazoxide choline) was developed for the treatment of hyperphagia, extreme hunger that cannot be satisfied, in people with Prader-Willi syndrome, a genetic condition that affects growth, development and behaviour.
The application for a new use of Pluvicto (lutetium (177Lu) vipivotide tetraxetan), a medicine to treat cancer of the prostate, to treat adults with PSMA-positive mCRPC who have no or mild symptoms, after their cancer has worsened despite treatment with a hormone blocking medicine, was withdrawn.
Question-and-answer documents on the withdrawal of these two applications are available in the grid below.
Other updates
The CHMP has finalised its assessment of an application to extend the use of Opdualag (nivolumab/relatlimab) to include the treatment of advanced melanoma, a type of skin cancer that has spread or cannot be surgically removed, with PD-L1 levels of 1% or higher. PD-L1 is a protein produced by some cancer cells. Although EMA did not recommend this use, it agreed that relevant data submitted with the application be included in the medicine’s product information, so that healthcare professionals have access to up-to-date data on the effects of Opdualag in patients with advanced melanoma with PD-L1 levels below 1%.
For more information, see the question-and-answer document in the grid below.
Agenda and minutes
The agenda of the April 2026 CHMP meeting is published on EMA's website. Minutes of the meeting will be published in the coming weeks.
CHMP statistics
Key figures from the April 2026 CHMP meeting are represented in the graphic below.
CHMP statistics: Text version
April 2026 statistics - monthly and cumulative figures for CHMP opinions and withdrawn applications:
• 5 positive opinions on new medicines: 1 new non-orphan medicine, 2 orphan medicines, 1 biosimilar and 1 generic, hybrid or informed consent medicine. Total in 2026: 28
• 0 negative opinions on new medicines. Total in 2026: 0
• 10 positive opinions on extensions of therapeutic indication. Total in 2026: 39
• 1 withdrawn applications for new medicines. Total in 2026: 3
Positive recommendations on new medicines
Cenrifki
INN tolebrutinib
Marketing authorisation applicant Sanofi Winthrop Industrie
Therapeutic indication Treatment of non-relapsing secondary progressive multiple sclerosis (nrSPMS) in adults
More information Cenrifki: pending EC decision
Itvisma
INN onasemnogene abeparvovec
Marketing authorisation applicant Novartis Europharm Limited
Therapeutic indication Treatment of 5q spinal muscular atrophy (SMA)
Orphan designation This medicine was designated an orphan medicine
More information Itvisma: pending EC decision
Redemplo
INN plozasiran
Marketing authorisation applicant Arrowhead Pharmaceuticals Ireland Limited
Therapeutic indication Treatment of familial chylomicronaemia syndrome (FCS)
Orphan designation This medicine was designated an orphan medicine
More information Redemplo: pending EC decision
News New medicine to reduce triglycerides in adults with familial chylomicronaemia syndrome
Positive recommendations on new biosimilar medicine
Rexatilux
INN ranibizumab
Marketing authorisation holder Intas Third Party Sales 2005 S.L.
Therapeutic indication Treatment of adults with neovascular (wet) age-related macular degeneration (AMD), visual impairment and other retinopathies
More information Rexatilux: pending EC decision
Positive recommendations on new generic medicine
Palbociclib Viatris
INN palbociclib
Marketing authorisation holder Viatris Limited
Therapeutic indication Treatment of breast cancer
More information Palbociclib Viatris: pending EC decision
Positive recommendations on extensions of therapeutic indications
Agamree
INN vamorolone
Marketing authorisation holder Santhera Pharmaceuticals (Deutschland) GmbH
More information Agamree: pending EC decision
Aquipta
INN atogepant
Marketing authorisation holder Abbvie Deutschland GmbH & Co. KG
More information Aquipta: pending EC decision
Comirnaty
INN COVID-19 mRNA vaccine
Marketing authorisation holder BioNTech Manufacturing GmbH
More information Comirnaty: pending EC decision
Crysvita
INN burosumab
Marketing authorisation holder Kyowa Kirin Holdings B.V.
More information Crysvita: pending EC decision
Inaqovi
INN decitabine / cedazuridine
Marketing authorisation holder Otsuka Pharmaceutical Netherlands B.V.
More information Inaqovi: pending EC decision
Opdivo
INN nivolumab
Marketing authorisation holder Bristol-Myers Squibb Pharma EEIG
More information Opdivo: pending EC decision
Privigen
INN human normal immunoglobulin
Marketing authorisation holder CSL Behring GmbH
More information Privigen: pending EC decision
Skyrizi
INN risankizumab
Marketing authorisation holder Abbvie Deutschland GmbH & Co. KG
More information Skyrizi: pending EC decision
Venclyxto
INN venetoclax
Marketing authorisation holder Abbvie Deutschland GmbH & Co. KG
More information Venclyxto: pending EC decision (EMA/VR/0000322237) Venclyxto: pending EC decision (EMA/VR/0000322240)
Withdrawal of initial marketing authorisation application
Viokat
INN diazoxide choline
Marketing authorisation applicant Soleno Therapeutics Europe Limited
Therapeutic indication Treatment of adult and paediatric patients with Prader-Willi syndrome (PWS)
Orphan designation This medicine was designated an orphan medicine
More information Viokat: questions and answers
Withdrawal of application to change the marketing authorisation
Pluvicto
INN lutetium (177Lu) vipivotide tetraxetan
Marketing authorisation holder Novartis Europharm Limited
More information Pluvicto: questions and answers
Other updates
Questions and answers on the outcome of assessment on use of Opdualag in the treatment of advanced melanoma
Adopted Reference Number: EMA/89906/2026
English (EN) (141.78 KB - PDF)
First published: 24/04/2026
View
Recommendations on eligibility to PRIME scheme - Adopted at the CHMP meeting of 20-23 April 2026
Adopted Reference Number: EMA/93274/2026
English (EN) (212.61 KB - PDF)
First published: 08/05/2026
View
Scientific advice and protocol assistance adopted during the CHMP meeting 20-23 April 2026
Adopted Reference Number: EMA/118426/2026
English (EN) (242.41 KB - PDF)
First published: 21/05/2026
View
Please note that not all documents associated with this page are published at the same time. This page is updated with new documents as soon as they become available. Therefore, users are asked to check the page regularly.
Related content
• Committee for Medicinal Products for Human Use (CHMP): 20-23 April 2026
• Committee for Medicinal Products for Human Use (CHMP)
News
• New medicine to reduce triglycerides in adults with familial chylomicronaemia syndrome
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Related medicine information
• Cenrifki
• Itvisma
• Redemplo
• Palbociclib Viatris
• Rexatilux
• Agamree
• Aquipta
• Inaqovi
• Opdivo
• Privigen
• Skyrizi
• Venclyxto
• Opdualag
• Pluvicto
• Viokat
• Comirnaty
Reproduced from EMA News · view original · public domain or open-licensed government work