1. Scrip
  2. >>Business

Sarepta Smacked By FDA's Curiously Timed Compassionate Use Revisions

 
• By Donna Young

Sarepta Therapeutics Inc. investors panicked over the FDA's June 2 release of revised expanded access guidances – worried the action was in preparation for the agency to reject the company's Duchenne muscular dystrophy drug eteplirsen, whose verdict was curiously delayed last week.

While the FDA insisted there was absolutely no connection to its June 2 release of its revised expanded access guidances, which include the finalization of a streamlined process for doctors to request "compassionate use" of investigational medicines, and the curiously timed delay last week of the agency's decision on Sarepta Therapeutics Inc.'s experimental Duchenne muscular dystrophy (DMD) treatment eteplirsen, Wall Street was unconvinced.

Indeed, most analysts and investors interpreted the FDA's updates to its expanded access program as having direct implications for Sarepta...

Welcome to Scrip

Create an account to read this article

Already a subscriber?

More from Business

Bristol Appoints AstraZeneca’s Massacesi As Next CMO

 
• By Jessica Merrill

Chief Medical Officer Samit Hirawat will step down to pursue new career opportunities, the company said.

Boehringer Ingelheim Expects Two Key Approvals In Second Half Of 2025

 
• By Andrew McConaghie

The privately held German pharma firm is hoping to bring a new generation of pulmonary fibrosis and cancer drugs to market but their commercial success is not yet a certainty.

Deal Watch: Novartis Hopes To Ace Inflammatory Disease With Matchpoint Tie-Up

 
• By Joseph Haas, Lisa Takagi, and Manas Mishra

Plus deals involving Lilly/Gate Bioscience, Concentra/iTeos, Nicox/Kowa, Ajax/Schrodinger, I-Mab/Bridge Health, Otsuka/Cantargia, JCR/Acumen and more.

Viking Rapidly Advancing Oral And Injectable VK2735 For Obesity

 
• By Joseph Haas

In its Q2 update, Viking reported that Phase III trials of subcutaneous VK2735 are under way, and that it expects Phase II data for an oral formulation before year’s end.

More from Scrip

Deal Watch: Novartis Hopes To Ace Inflammatory Disease With Matchpoint Tie-Up

 
• By Joseph Haas, Lisa Takagi, and Manas Mishra

Plus deals involving Lilly/Gate Bioscience, Concentra/iTeos, Nicox/Kowa, Ajax/Schrodinger, I-Mab/Bridge Health, Otsuka/Cantargia, JCR/Acumen and more.

More Pressure on Elevidys As The EU’s CHMP Says ‘No’

 
• By Alexandra Shimmings

Just days after Roche stopped shipments of the DMD gene therapy following safety concerns raised by two patient deaths, its EU approval application has hit a block.

Executives On The Move: Three New CEOs Among This Week’s Changes

Recent moves in the industry include changes at the top at Trogenix and Quetzal Therapeutics, plus Synendos Therapeutics gains a new CMO from Roche.