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Accelerated Approval After Eteplirsen: A Lowered Bar Or A Unique Event?

 
• By Sue Sutter

What CDER Director Woodcock saw as a flexible and reasonable approach to approval of Sarepta's muscular dystrophy drug, ODE I Director Unger viewed as a threat to the substantial evidence standard.

FDAs internal disagreement over the adequacy of Sarepta Therapeutics Inc.'s efficacy evidence for Exondys 51 (eteplirsen) evolved into a debate over the intent, utility and future of the accelerated approval regulatory pathway.

Office of Drug Evaluation I Director Ellis Unger and others within the agency worried that granting eteplirsen accelerated approval for...

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More from Approvals

America Next: Most Novel Agents With July Goal Dates Have Been Approved Overseas

 
• By Bridget Silverman

The US FDA will not be in its usual position as the first regulator to approve novel drugs, with only one of the six novel candidates on the July user fee calendar seeking its first approval worldwide in the US

EU Pharma Reform: Lawyers Unpack Diverging Visions For Innovation

 
• By Eliza Slawther

There are now three draft versions of the legislative text that will overhaul the framework governing pharmaceuticals in the EU. Lawyers explain how the approaches proposed by the European Commission, European Parliament and Council of the EU differ and highlight implications for industry.

New EU Filings

 
• By Neena Brizmohun

Linerixibat, GSK's treatment for cholestatic pruritus in patients with primary biliary cholangitis, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

EMA Nod For First MASH Drug Rezdiffra, Cell Therapy Zemcelpro And 11 Others

 
• By Eliza Slawther

Madrigal Pharmaceuticals’ Rezdiffra is on track to become the first medicine approved in the EU for non-cirrhotic metabolic dysfunction-associated steatohepatitis after the European Medicines Agency recommended that it be granted conditional marketing authorization.

More from Product Reviews

New CDC Vaccine Panel Chair Vows To ‘Rebuild Public Trust’ With Evidence-Based Medicine

 
• By Sue Sutter

The newly reconstituted Advisory Committee on Immunization Practices will create new work groups to examine the cumulative impact of vaccine schedules and reexamine some older vaccines.

Cassidy Confirms Agreement With Kennedy Did Not Include Maintaining CDC Vaccine Advisors

 
• By Sarah Karlin-Smith

Sen. Bill Cassidy and HHS Secretary Robert F. Kennedy Jr. agree the disbanding of ACIP did not violate their agreement, but the perception that Kennedy is not following the spirit of the pledge could be more important long-term.

EU CHMP Opinions And MAA Updates

 
• By Neena Brizmohun

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.