FDA approves Pfizer’s 1st gene therapy

Pfizer’s first FDA-approved gene therapy is one of the most expensive medications in the U.S.

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On April 26, Beqvez (fidanacogene elaparvovec-dzkt) won approval to treat some adults with moderate-to-severe hemophilia B. The drugmaker set the list price at $3.5 million, a company spokesperson told CNBC. Another hemophilia B gene medication, Hemgenix, costs the same. 

In a phase 3 study of 45 patients, researchers found a decrease in annualized bleeding rates among those who received the medication, which was well-tolerated.

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