Best Read Gene Therapy Stories of 2023
FDA Approves First Cellular Therapy for Type 1 Diabetes
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.
First Gene Therapy for Duchenne Priced at $3.2 Million
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.
State Medicaid Programs Look to Value-Based Contracts for Gene Therapies
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.
FDA Approves Two Gene Therapies for Sickle Cell Disease
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.
FDA Approves $2.9 Million Gene Therapy for Hemophilia A
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.
Navitus to Offer Unbranded Stelara Biosimilar, Remove Stelara from Formulary
March 13th 2025Lumicera Health Services, Navitus’ specialty pharmacy, has made a deal with Teva to offer an unbranded biosimilar that they estimate will save $112,000 and $336,000 per patient per year. Navitus will remove Stelara from formulary on July 1, 2025.
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FDA Approves Neffy 1 mg Nasal Spray for Pediatric Patients
March 6th 2025Neffy 1 mg is now approved by the FDA to treat pediatric patients who weigh 33 to 65 lbs. Neffy was first FDA-approved as a 2 mg dose in August 2024 for the emergency treatment of anaphylaxis in children and adults weighing at least 66 lbs.
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