In a world first, regulators in the U.K. approved Crispr Therapeutics' gene-editing treatment for two blood diseases on Thursday. CRSP stock surged on the news.
The drug formerly known as exa-cel will sell under the brand name Casgevy following the approval. The Medicines and Healthcare products Regulatory Agency approved Casgevy for people age 12 and older with sickle cell disease or beta thalassemia.
"Today is a historic day in science and medicine," Vertex Pharmaceuticals Chief Executive Reshma Kewalramani said in a statement. Vertex partnered with Crispr on the drug. "This authorization of Casgevy in Great Britain is the first regulatory authorization of a CRISPR-based therapy in the world."
On today's stock market, CRSP stock jumped 5.3% to 59.22. Vertex shares sank 1.8% to 343.
CRSP Stock: 2,000 UK Patients
Specifically, the MHRA signed off on Casgevy for patients with sickle cell disease who experience severe episodes of pain called vaso-occlusive crises, and beta thalassemia patients who require frequent blood transfusions. These patients must also be unable to find a stem cell donor.
Crispr estimates there are 2,000 patients in the U.K. who fit this bill.
The approval is conditional, meaning it's contingent upon continued testing and must be renewed every year. The Food and Drug Administration is due to make its decision on the use of exa-cel in treating sickle cell disease on Dec. 8. The FDA is also expected to make a decision on the drug's use for beta thalassemia on March 30.
"While this approval comes in line with our expectations, the approval remains a historic milestone for gene-editing as a whole, as this technology enters the commercial market in earnest," Leerink Partners analyst Mani Foroohar said in a written statement.
He has an outperform rating on CRSP stock.
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