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Crispr Therapeutics AG
CRSP,
and Vertex Pharmaceuticals Inc.
VRTX,
on Friday got U.S. regulatory approval for a treatment for sickle-cell disease based on the transformative gene-editing technology Crispr.
The U.S. Food and Drug Administration approved the treatment, known as Casgevy or exa-cel, for treatment of sickle-cell disease in patients age 12 and older who have recurrent issues with affected red blood cells blocking blood flow.
Casgevy last month got the world’s first regulatory approval for a Crispr-based medicine when a U.K. regulator approved it for treatment of sickle-cell disease. The medicine edits a faulty gene in people with sickle-cell disease, prompting the production of functioning hemoglobin that carries oxygen to the tissues.
The FDA on Friday also approved Bluebird bio Inc.’s
BLUE,
Lyfgenia, a cell-based gene therapy for sickle-cell disease.
“These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health,” Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement.
Sickle-cell disease is an inherited disorder that affects red blood cells, causing them to become stiff and sticky and disrupting delivery of oxygen to the tissues. Roughly 100,000 people in the U.S. have the condition, which disproportionately affects Black people, according to estimates from the Centers for Disease Control and Prevention.
Casgevy won’t be cheap, analysts say. The medicine would be cost-effective if priced at up to $2.05 million, the Institute for Clinical and Economic Review said in an August report, noting that “potentially curative gene therapies should and will command a high price.” But sickle-cell disease “heavily affects the American descendants of those who were forcibly brought here as slaves. As such, the U.S. government and U.S. payers have special obligations to ensure access” to the new treatment, ICER chief medical officer Dr. David Rind said in a statement when the report was issued.
A Vertex spokesperson said the price of exa-cel will be announced after its approval.
Stifel analysts said in a report Thursday, ahead of the FDA’s decision on exa-cel, that the agency’s approval could be meaningful for other Crispr-focused companies. Editas Medicine Inc.’s
EDIT,
experimental cell therapy EDIT-301, for example, “shares many similarities” with exa-cel, the analysts wrote, and the FDA action on exa-cel could be seen as cutting risks for that therapy.
Crispr Therapeutics shares were down 9% Friday and have climbed 56% in the year to date, while Bluebird bio shares gained more than 7% Friday and have dropped 25% so far this year.
