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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...

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More from Review Pathways

Brazil & Mexico Bet On Regulatory Reliance To Improve Pharmacovigilance & Access To Medicines

 
• By Francesca Bruce

Two of Latin America’s biggest regulators have signed a memorandum of understanding to deepen cooperation and improve access to medicines.

Project Orbis: 2025 Shaping Up To Be A Strong Year For Canadian Approvals

 
• By Eliza Slawther

Health Canada has approved four new drugs and 12 label variations so far this year via Project Orbis, a combined figure that already exceeds the annual approval figures for the two previous years. The agency explains the factors that impact annual approvals and highlights the pathway’s benefits.

Project Orbis: Annual UK Approvals Down Since 2022, But MHRA Touts Advantages

 
• By Eliza Slawther

The UK MHRA has emphasized the merits of the Project Orbis scheme, which has resulted in 12 new drug approvals and 14 indication extensions in the country so far. However, overall annual approval figures were lower in 2023 and 2024 than in the years prior.

‘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.

More from Pathways & Standards

Navigating Rare Disease Submissions: Minoryx Stresses Early EMA Engagement

 
• By Manas Mishra

Minoryx explained to Pink Sheet why it believes its resubmission for leriglitazone stands a stronger chance of approval, and highlighted the value of early dialogue with the regulators when it comes to rare diseases, where the understanding of the natural history and outcomes is often limited.

Oncology: Plan Ahead To Rule Out Harm When Survival Not The Endpoint, US FDA Says

 
• By Michael McCaughan

The FDA recommended sponsors plan ahead for potential issues if overall survival is not feasible as a primary or secondary efficacy endpoint in clinical trials and must be analyzed as a safety outcome.

Selective Safety Data Collection In Clinical Trials: Adoption Lags Despite Benefits, US FDA Says

 
• By Manas Mishra

Lack of awareness is limiting use of guidelines that simplify safety reporting for some late-stage trials, FDA says, while also highlighting wins. Boehringer experienced mixed results, while Novartis, Lilly, and Merck were more successful, and Roche should have probably tried, white paper suggests.