In June, the FDA approved Lantidra (donislecel), the first allogeneic (donor) pancreatic islet cellular therapy to treat patients with type 1 diabetes. In two small studies, patients were able to be free from insulin injections for a year or more.
Elevidys is a one-time therapy that delivers to muscle a gene that codes for a shortened, functional form of dystrophin, which is mutated in Duchenne muscular dystrophy.
Magellan Rx Management has begun providing a multi-state solution in which they negotiate with drug manufacturers for value-based contracts for high-cost gene and cell therapies.
The FDA has approved the gene therapies Lyfgenia and Casgevy to treat patients with sickle cell disease. Casgevy is the first FDA-approved gene therapy to use the CRISPR gene editing technology.
BioMarin is offering an outcomes-based warranty that will reimburse payers up to 100% of the cost if a patient does not respond to Roctavian.