Internal FDA documents reveal a familiar story for Sarepta
The unexpected approval of Vyondys 53 followed an FDA power struggle, and turned on a vague commitment from Sarepta, it has emerged.
Morphosys gets a major tafasitamab endorsement
Incyte hands across $750m up front and buys $150m of Morphosys equity for rights to the anti-CD19 project.
Sarepta makes history with Roche deal
A bidding war has netted Sarepta the largest-ever licensing deal for a cell or gene therapy. Can Roche make it work?
Gene therapy clinical holds take centre stage
An analysis of company disclosures reveals a sharp rise in US FDA-imposed halts on clinical work involving gene therapies.
A chance for the US regulator to show its teeth, at Amag’s expense
An advisory panel votes to pull Amag’s Makena from the market, but will the US regulator follow through?
Wave takes a different tack from Sarepta in Duchenne
Wave will soon report key early-stage data with its exon-skipping DMD candidate suvodirsen but, unlike Sarepta, the group is starting its confirmatory trial before…
Zogenix gets an unwelcome distraction from Fintepla
Spending $250m on Modis gives Zogenix a new rare disease niche, but the timing and the target both look odd.
Astra could use a voucher to cement roxadustat’s head start
Spending $95m to speed up the anaemia project’s path to approval could be worth the investment for Astrazeneca.
The Sarepta fairy tale takes a worrying turn
A complete response letter for Sarepta’s follow-on Duchenne therapy is a big blow for the company, but in hindsight was the FDA’s decision actually that surprising?
