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The Eteplirsen Approval: Former FDA Officials Weigh In On The Science

 
• By Ramsey Baghdadi

A former CDER director, office director, division director, and supervisory reviewer agreed to comment for the record on the merits of the science that formed the basis of FDA’s accelerated approval of the Duchenne Muscular Dystrophy therapy Exondys 51.

FDA’s decision to grant accelerated approval to Sarepta’s DMD drug Exondys 51 (eteplirsen) has generated divergent opinions on what the approval means to the agency standards for approval and to the relationships between the different review levels within the Center for Drug Evaluation and Research.

Further coverage of those issues can be found in the sidebars, but behind them all is the important fundamental assessment...

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US FDA Maintaining Approval Output Despite Losing Staff

 
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Reviewers were not targeted by the FDA reduction in force, but increasing attrition and difficulties hiring new staff still do not seem to have impacted new and generic drug approval totals.

Precigen’s Papzimeous Approval Allows US FDA CBER Director To Emphasize Flexibility

 
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Inside Elamipretide’s CRL: Clinical And Surrogate Evidence Deficiencies, And A Path Forward

 
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The Pink Sheet takes an exclusive look at the complete response letter, which shows the information on application deficiencies that could become widely available if FDA leaders succeed in releasing CRLs in real time.

Stealth Pushes for Faster US FDA Review Of Barth Syndrome Drug As Funds Dwindle

 
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The Pink Sheet viewed Stealth's complete response letter and other documents indicating the FDA appears willing to grant accelerated approval to elamipretide before a confirmatory trial begins enrolling. But the company says it needs a two-month review of the NDA resubmission to survive.

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UK Tests Risk-Proportionate Review Of Substantial Clinical Trial Amendments

 
• By Vibha Sharma

Sponsors running clinical trials in the UK can get a decision on their applications to make substantial but low-risk amendments to their studies within 14 days under a pilot that is testing a streamlined pathway ahead of new clinical trial regulations coming into force in April 2026.

‘We Need To Move With The Times’: Cell & Gene Therapy Catapult CEO On UK’s Regulatory Overhaul

 
• By Eliza Slawther

The UK drug regulator has introduced several changes in recent months, such as the introduction of a decentralized manufacturing regulation. Matthew Durdy, CEO of the Cell and Gene Therapy Catapult, explains what these changes mean for the cell and gene industry.

Boosting EU Clinical Trials: EMA Opens Dialogue With Global CRO Group

 
• By Vibha Sharma

The European Medicines Agency’s inaugural bilateral meeting with the Association of Clinical Research Organizations underscores ACRO’s commitment to advancing innovation and its presence in the EU clinical trials ecosystem.