Update for October 3, 2022

Regulatory Update
 
The FDA approved the combination of sodium phenylbutyrate and taurursodiol (Relyvrio, Amylyx Pharmaceuticals), on 9/29/2022, to slow the functional decline in Amyotrophic Lateral Sclerosis (ALS). Amylyx set WAC for Relyvrio at $158,000 per year. This is slightly less than the cost of intravenous edaravone, but much higher than ICER’s recommended health-benefit price benchmark range of $9,100 to $30,700.
 
The FDA approved futibatinib (Lytgobi, Taiho Oncology), on 9/30/2022, for the treatment of unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 gene fusions or other rearrangements.
 
The FDA accepted the NDA for leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome and assigned a PDUFA date of 3/29/23.
 
BioMarin resubmitted a BLA for valoctocogene roxaparvovec to treat severe hemophilia A with two outcomes from the Phase III GENEr8-1 trial and five years data form a Phase I/II trial.
 
Sarepta submitted a BLA for delandistrogene moxeparvovec for the treatment of ambulant Duchenne muscular dystrophy in September 2022.
 
The EMA accepted the MAA for fezolinetant for the treatment of moderate to severe vasomotor symptoms associated with menopause.