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How Accelerated Approval Works – And How It Doesn't

 
• By Derrick Gingery

PTC Therapeutics argues FDA did not apply the same standard to Translarna in Duchenne muscular dystrophy as previous DMD products, but FDA says accelerated approval was not possible because of negative efficacy data.

FDA Advisory Committee Feature image

US FDA officials are refusing to allow PTC Therapeutics Inc.'s Translarna (ataluren) to use the precedent for accelerated approval in Duchenne muscular dystrophy that was created by the controversial approval of Sarepta Therapeutics Inc.'s Exondys 51 (eteplirsen).

Agency officials told the Peripheral and Central Nervous System Drugs Advisory Committee Sept. 28 that because the efficacy studies submitted were negative or problematic, the pathway could not be used.

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US FDA’s Prasad: ‘We Will Always Embrace Surrogate Endpoints’

 
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The new CBER director, once best known in the pharma world for criticizing accelerated approval, committed to expediting cancer drugs with surrogate endpoints.

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The agency said it will consider knee extensor muscle strength as a potential intermediate clinical endpoint to support accelerated approval, but Stealth CEO Reenie McCarthy said announcing it via complete response letter is confusing and inefficient.

EMA Touts Faster Global Post-Approval Changes Via Regulatory Reliance

 
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The European Medicines Agency says that a regulatory reliance pilot that it is supporting to speed up assessments of post-approval changes appears to be accelerating evaluation timelines in non-EU countries.

EMA Says Digital Transformation A ‘Fundamental Shift’ In Meeting Regulatory Challenges

 
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Sponsors can expect faster evaluation processes for key medicines and greater support in mitigating medicines supply shortages this year as part of the European Medicines Agency’s digital transformation overhaul.

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New EU Approvals

 
• By Neena Brizmohun

The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include two new products, one of which is Vyjuvek, Krystal Biotech's advanced therapy for treating dystrophic epidermolysis bullosa.

EMA Says Digital Transformation A ‘Fundamental Shift’ In Meeting Regulatory Challenges

 
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EMA: ‘Sad To See’ Member States Against Patient Engagement Under EU Pharma Reform

 
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The European Medicines Agency’s chief medical officer Steffen Thistrup argued that patients are able to understand the “complex regulatory and scientific issues” that the EMA committees discuss when they assess drugs.