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First Oral Treatment For Spinal Muscular Atrophy Gets EMA Nod

Two Of Six Drugs Recommended For EU-Wide Use Have Orphan Designation

 
• By Vibha Sharma

New treatments for advanced endometrial cancer and hereditary angioedema are among six drugs that have just been recommended for EU-wide approval. Harmonized EU advice has also been issued in relation to an investigational antibody combination for treating COVID-19.

Surgical doctor looking at radiological spinal x-ray film for medical diagnosis on patient’s health on spine disease, bone cancer illness, spinal muscular atrophy, medical healthcare concept
Current treatments for SMA involve frequent clinic visits or invasive procedures • Source: Shutterstock

Roche’s orphan drug Evrysdi (risdiplam) for treating certain types of spinal muscular atrophy (SMA) is among six medicines recommended for EU-wide approval this month by the European Medicines Agency’s human medicines evaluation committee, the CHMP.

If approved, Evrysdi will become the first oral, at-home treatment for SMA in the EU

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More from Approvals

New EU Filings

 
• By Neena Brizmohun

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Potency Assay Issues Delayed US FDA Approval Of Mesoblast’s Ryoncil For Years

 
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Mesoblast’s Ryoncil: US FDA Changed Its Mind On Need For A Randomized Trial

 
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Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.

Ryoncil Chronology: Three Review Cycles, Two CRLs, One Dispute Resolution Request

 
• By Sue Sutter

The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.

More from Product Reviews

FDA Adcomms Are Back: Four Cancer Drugs, COVID-19 Vaccine Formulations To Get Reviews

 
• By Sue Sutter and Bridget Silverman

The Oncologic Drugs Advisory Committee will meet for two days in mid-May, followed by a Vaccines and Related Biological Products Advisory Committee’s review of the 2025-2026 COVID-19 vaccine formulation.

Unlocking Opportunities: How To Engage With The EMA On Animal Testing Alternatives

 
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Opioid Safety: US FDA Hears Mixed Messages On Adding Posmarketing Studies To Labeling

 
• By Sue Sutter

Some advisory committee members said adding quantitative data on misuse, abuse, opioid use disorder and overdose to labeling would be helpful, but others worried the two epidemiological studies were not sufficiently generalizable to a broader population.