Update for March 23, 2022

Regulatory Update
 
The FDA approved the combination of nivolumab and relatlimab (Opdualag, Bristol-Myers Squibb), on 3/18/2022, for the treatment of unresectable or metastatic melanoma.
 
The FDA approved ganaxolone (Ztalmy, Marinus pharmaceuticals), on 3/18/2022, for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder. ganaxolone is expected to be available in July following scheduling by the DEA.
 
Phantom submitted an NDA to the FDA for the use of vonoprazan to treat all grades of erosive esophagitis.
 
Sodium phenylbutyrate/taurursodiol (AMX0035) is available through an Expanded Access Program (NCT05286372).
 
Announced Research Updates
 
Astellas announced interim results for the Phase III, fezolinetant MOONLIGHT 1 trial.
 
Allena announced interim results from the URIROX-2 trial which lead to the discontinuation of reloxaliase development.
 
Amicus announced 36-month results of treatment with cipaglucosidase alfa/miglustat in patients with Pompe disease.
 
Published Research Updates
 
The results of EMERGE and ENGAGE were published in a single article in the Journal of Prevention of Alzheimer’s Disease. The data presented in the article is the same as has been previously announced. The article did not address limitations identified in the FDA statistical analysis of the trials nor add any insights.
 
One-year results were published for the GENEr8-1 trial, which evaluated a single dose of valoctocogene roxaparvovec in the treatment of severe hemophilia A.