Update for May 24, 2023

Regulatory Update
 
The FDA approved beremagene geperpavec (Vyjuvek, Krystal Biotech), on 5/19/2023, for the treatment of dystrophic epidermolysis bullosa.
 
The FDA approved epcoritamab (Epkinly, Genmab & AbbVie), on 5/19/2023, for the treatment of relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy. Epcoritamab was approved with a boxed warning regarding Cytokine Release Syndrome and Immune Effector Cell-Associated Neurotoxicity Syndrome 
 
An FDA review of RSVpreF for the prevention of lower respiratory tract disease caused by RSV in infants, when administered to pregnant mothers, found the vaccine to be efficacious and estimated vaccine efficacy to be 57.1% within 90 days of birth and 51.3% during the first 180 days. RSVpreF was generally safe and had an adverse effect profile similar to placebo (16.2% and 15.2%). It was noted that while there was a numerically higher occurrence of premature delivery, the difference was not significant (5.6% vs 4.7%). The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted 14 to 0 on effectiveness and 10 to 4 on safety for RSVpreF for the prevention of lower respiratory tract disease caused by RSV in infants, when administered to pregnant mothers. Members who voted no for safety were concerned about the higher incidence of premature deliveries.
 
Elevar Therapeutics submitted an NDA for apatinib plus camrelizumab for the treatment of hepatocellular carcinoma.
 
The FDA accepted the BLA for TAK-755 for the treatment of congenital thrombotic thrombocytopenic purpura, which suggests a PDUFA date on 11/16/2023.
 
Mustang Bio announced it would delay initiation of Phase III trials for MB-107 and MB-207 until after a research partner completes a trial evaluating a modified version of the current lentiviral vector. Mustang Bio will initiate the Phase III trials utilizing the safest vector.
 
The FDA granted isaralgagene civaparvovec a Fast Track Designation for the treatment of Fabry disease.