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Confirmatory Studies For Rare Diseases Shouldn’t Need To Start Before Accelerated Approval, Stakeholders Argue

 
• By Derrick Gingery

Advocates and sponsors looking to shape implementation of US FDA’s new mandate want to ensure firms are not required to make significant investments in surrogate endpoints before they are validated.

clinical trial use
A blanket mandate to start confirmatory trials when accelerated approval is granted could force significant investment in surrogate endpoints that are not accepted. • Source: Shutterstock

BOSTON – Having a confirmatory study ongoing at the time of an accelerated approval may be a laudable goal, but some stakeholders worry a blanket requirement could hinder rare disease development.

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European Regulator Deals Blow For Lilly’s Alzheimer’s Drug Kisunla

 
• By Francesca Bruce

Eli Lilly’ will request a re-examination after the European Medicines Agency declined to recommend its Alzheimer’s disease drug Kisunla for EU approval.

EMA’s Newly Qualified AI Tool To Boost MASH Market Dynamics

 
• By Neena Brizmohun

The European Medicines Agency’s qualification of the AIM-NASH tool is said to signify a major advancement for clinical trials for metabolic dysfunction-associated steatohepatitis. The market size for MASH treatments is expected to grow substantially in the coming years.

EMA Considers Whether Intrathecal Zolgensma Deserves Fast Tracking

 
• By Neena Brizmohun

Novartis is planning to file EU and US marketing applications for an intrathecal formulation of its spinal muscular atrophy gene therapy, Zolgensma, in H1 2025.