New hope for Sickle Cell Anaemia patients as FDA gives nod to Casgevy, Lyfgenia gene therapies

The US Food and Drug Administration (FDA) has given the green light to two new gene therapies for sickle cell disease.One of them, called Casgevy, is a treatment that uses the CRISPR gene editing technology, which won the Nobel Prize. The other approved therapy is Lyfgenia, developed by bluebird bio. Both treatments are meant for individuals aged 12 and above.

Casgevy is developed by Vertex Pharmaceuticals and CRISPR Therapeutics and comes with 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, media reports said.

Sickle Cell Disease (SCD) is an inherited blood disorder that causes pain and complications due to the production of flawed, sickle-shaped hemoglobin. This impairs the proper transport of oxygen by red blood cells to the body's tissues. It affects around 100,000 people in the United States, primarily among the Black population.

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The approval is a crucial 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.

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In clinical trials, both gene therapies demonstrated effectiveness in reducing painful episodes in patients with sickle cell disease. Casgevy, based on CRISPR technology, uses molecular "scissors" to correct faulty genes. On the other hand, Lyfgenia works by introducing modified genes into the body through disabled viruses.

Cancer risks?

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.

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While both therapies show promise, data on their long-term effects is limited. 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.

(With inputs from agencies)