The US Food and Drug Administration (FDA) has granted approval for two new gene therapies targeting sickle cell disease. One of them, named Casgevy, utilizes the CRISPR gene editing technology, while the other, Lyfgenia, is developed by bluebird bio. Both therapies are designed for individuals aged 12 and above.
Casgevy, created by Vertex Pharmaceuticals and CRISPR Therapeutics, carries a price tag of $2.2 million, while bluebird’s Lyfgenia is priced at $3.1 million. These are considered one-time treatments and are expected to be available in early 2024.
Sickle Cell Disease (SCD) is an inherited blood disorder causing pain and complications due to the production of flawed, sickle-shaped hemoglobin, hindering the proper transport of oxygen by red blood cells to the body’s tissues. It predominantly affects the Black population, impacting around 100,000 people in the United States.
The approval marks a significant advancement for the sickle cell disease community, which, according to Robert Brodsky, President of the American Society of Hematology, has historically faced neglect and underfunding.
In clinical trials, both gene therapies demonstrated effectiveness in reducing painful episodes in patients with sickle cell disease. Casgevy, based on CRISPR technology, employs molecular “scissors” to correct faulty genes. Lyfgenia, on the other hand, introduces modified genes into the body through disabled viruses.
However, the FDA has issued a warning on Lyfgenia’s label regarding the risk of blood cancer after two patients in bluebird’s trial developed acute myeloid leukemia. The FDA will closely monitor potential side effects.
While both therapies show promise, data on their long-term effects is limited. To address this, the companies plan to assess potential long-term safety risks through a 15-year follow-up study after approval. Patients will be monitored throughout their lives for cancer malignancies.
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