FDA Approves Game-Changing Gene Therapies for Sickle Cell Disease
TL;DR
The U.S. FDA has granted approval for two gene therapies, Casgevy and Lyfgenia, for the treatment of sickle cell disease (SCD). Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first treatment in the U.S. to utilize CRISPR gene editing technology. SCD is an inherited blood disorder that affects around 100,000 people in the U.S., primarily Black individuals. Both therapies have shown promising results in reducing painful episodes in clinical trials. Casgevy has a list price of $2.2 million, while Lyfgenia is priced at $3.1 million. However, Lyfgenia's higher price and safety warning may impact its sales compared to Casgevy.
- US FDA approves two gene therapies for sickle cell disease Reuters
- F.D.A. Approves 2 Sickle Cell Treatments, One Using CRISPR Gene Editing The New York Times
- Gene editing could be a game changer for patients with sickle cell disease CBS Mornings
- A Sickle Cell Breakthrough Is Here. Now the Hard Part. Bloomberg
- Access barriers loom in Minnesota for blockbuster sickle cell therapy Star Tribune
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