Obstacle Thrown In Sarepta’s Path To Market As Gene Therapy Will Face AdComm After All
US FDA Looks To Probe Surrogate Endpoints
The route to market for Sarepta’s gene therapy candidate SRP-9001 for Duchenne muscular dystrophy has just got more complex after a U-turn by the US FDA. Its accelerated approval will now be scrutinized by outside experts – but the company remains confident in its data.
You may also be interested in...
No AdComm Puts Sarepta On Path To Blockbuster Success With Duchenne Gene Therapy
The US FDA’s waiving of an advisory committee is another promising signal that Sarepta's Duchenne gene therapy SRP-9001 could be approved on 29 May and set it up to become a bigger earner than Novartis’s Zolgensma.
Jemperli Results In Endometrial Cancer Help GSK Build ‘Backbone’ In Immunotherapy
Latecomer PD-1/L1 drug Jemperli has achieved impressive Phase III results in first line endometrial cancer, and GSK hopes it can “redefine” care, especially in dMMR patients. Rival Merck, however, has other ideas.
Pharming’s Portfolio Grows As Ultra-Rare Disease Drug Joenja Is Approved
The first ever approval for ultra-rare immunodeficiency disease APDS gives Pharming its second product on the market but patient diagnosis will be a major challenge.