Update for October 21, 2022

Regulatory Update
 
The FDA accepted the resubmitted BLA for BioMarin’s valoctocogene roxaparvovec, for the treatment of severe hemophilia A, and set a PDUFA date for 3/31/2023. 
 
The FDA delayed the PDUFA date for Biogen’s tofersen, by three months to 4/25/2023, and requested new information
 
The FDA granted Fast Track status to Nanoscope’s MCO-010 for the treatment of retinitis pigmentosa 
 
Pharming submitted an MAA for leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome.
 
CHMP recommended approval of: 

• Takeda’s dengue vaccine to prevent dengue fever
• Maralixibat to treat cholestatic pruritus in patients with Alagille syndrome
• Lutetium Lu 177 vipivotide tetraxeta to treat prostate cancer
• Spesolimab to treat generalized pustular psoriasis