1. Pink Sheet
  2. >>Product Reviews
  3. >>Drug Review Profiles

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...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

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

EU Biosimilar Filings, Opinions And Approvals

 
• By Neena Brizmohun

A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.

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.

Japan Gives Global-First Recommendations To Taisho’s Vornorexant, Santen’s Sepetaprost

 
• By Lisa Takagi

Japan gives world-first approval recommendations to two domestically originated drugs, for insomnia and glaucoma/ocular hypertension.