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Before Price Controversy, Emflaza Got Easy US Nod With Old But Good Enough Data

 
• By Bridget Silverman

Pink Sheet's monthly Drug Review Profile looks at Marathon's (now PTC's) Emflaza for Duchenne muscular dystrophy, and finds that before the steroid's price ignited controversy it received an unremarkable review – unremarkable, that is, for rare and serious illnesses. The US FDA accepted old and flawed trial data because signals of clinical benefit were consistently seen and safety profile was understood.

The US FDA's approval of Marathon Pharmaceuticals LLC's Emflaza (deflazacort) for Duchenne muscular dystrophy quickly set off a controversy about orphan drug pricing and value, but agency review documents show that the decision about the drug's efficacy and safety was anything but controversial.

"The benefits of deflazacort plainly outweigh its risks," Office of Drug Evaluation I Deputy Director Robert Temple concluded in his...

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More from Drug Review Profiles

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

 
• By Sue Sutter

The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.

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

 
• By Sue Sutter

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.

Testosterone, TRAVERSE, And A Label Change 15 Years In The Making

 
• By Bridget Silverman

The Pink Sheet drug review profile investigates the long and rigorous process behind the FDA’s recent relaxation of the cardiovascular safety warning for testosterone products.

More from Product Reviews

US FDA’s CRL Release More Incremental Than ‘Radical’ Transparency

 
• By Bridget Silverman

The transparency initiative collects CRLs already made public in approval packages on its drugs@fda site to post on the openFDA public portal.

US FDA Advisory Committee Supports Vaccine Safety, Scrutinizes AE Reporting Quality

 
• By Sue Sutter

The Pediatric Advisory Committee supported routine safety monitoring for three vaccines and 21 drugs, but the panel’s consumer representative said adverse event reporting needs improvement and FDA literature reviews should include lawsuits as well as social media.

US FDA Pediatric Adcomm To Examine HPV, Flu, Dengue Vaccines: No News Is Good News?

 
• By Sue Sutter

Pediatric Advisory Committee will weigh the FDA's recommendation that routine safety monitoring continue for Merck & Co's Gardasil and Sanofi's Fluzone and Dengvaxia despite a political climate that has been unkind to vaccines.