The group’s lead next-gen exon skipper beats a low bar set by Exondys, but adverse events loom large.
A US advisory panel has largely endorsed three anti-PD-(L)1 drugs, with voting apparently driven by the availability of alternatives.
May will see US FDA decisions for Apellis, Heron and Bristol Myers Squibb, while Provention Bio is set for a panel meeting.
A serious adverse event puts ADVM-022’s future in doubt, and it could hurt other eye disease gene therapy players too.
Amryt marches ahead, but gene therapies from Krystal, Abeona and Castle Creek are heading towards data readouts.