Dec 8, 2023 - Health

FDA approves CRISPR-based therapy for sickle cell disease

A researcher performs a CRISPR process at the Max-Delbrueck-Centre for Molecular Medicine in Berlin. Photo: Gregor Fischer/dpa (Photo by Gregor Fischer/picture alliance via Getty Images)

The Food and Drug Administration on Friday approved the first therapy based on CRISPR gene-editing technology to address the painful effects of sickle cell disease.

Why it matters: The technology could be applied beyond the estimated 100,000 Americans with sickle cell to other blood disorders like hemophilia, as well as certain cancers and infectious diseases.

  • But gene therapies are costly, with million-dollar price tags for some products, presenting huge challenges to employers, public health programs and other payers throughout the health system.
  • Morgan Stanley earlier this year projected the new sickle cell treatment could generate $19 billion in sales over the next decade.

Driving the news: The FDA approved Casgevy from Vertex Pharmaceuticals and CRISPR Therapeutics, a one-time treatment for patients 12 years and older.

  • The agency also approved Bluebird Bio's Lyfgenia gene therapy for patients 12 and older with sickle cell and a history of vaso-occlusive events.
  • The CRISPR therapy edits a patient's stem cells to produce high levels of fetal hemoglobin, which can offset the effects of defective hemoglobin that characterize the debilitating blood disorder.
  • An FDA advisory panel in November expressed confidence that the technology is ready for commercial use but said more research would be helpful.
  • One concern is whether the therapy could inadvertently target the wrong genetic sequence and increase the risk of a patient developing cancer.
  • The Institute for Clinical and Economic Review, which advises payers, estimated an appropriate price for Casgevy would be between $1.35 million and $2.05 million per patient.

What they're saying: "Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease," said Nicole Verdun, director of the Office of Therapeutic Products in the FDA's Center for Biologics Evaluation and Research.

  • "This CRISPR technique will be hugely expensive. But it will be life saving for a handful of people. And I don't think any employer is going to not cover that cost in some way," said Rob Andrews, CEO of the Health Transformation Alliance, which consists of major corporations like Marriott, Coca-Cola and American Express.

Catch up quick: Sickle cell disease is an inherited blood disorder that predominantly affects Black patients and alters the body's red blood cells so they have a sickle shape and don't bend or move easily.

  • It causes excruciating pain, and the disrupted blood flow can also lead to organ damage, stroke and eye problems. Until now, patients have had to turn to bone marrow transplants.
  • Despite its promise, the treatment can only be administered after grueling chemotherapy that destroys the patient's defective blood cells and may not be suitable for some older patients.

Tina Reed contributed.

Go deeper