Fierce Pharma November 7, 2023
After a long development road, the first CRISPR-based gene editing therapy recently cleared a FDA advisory committee and appears to be nearing its U.S. launch. Vertex Pharmaceuticals, awaiting the potential approval, is busy laying the groundwork for the med’s debut.
Exa-cel is slated for an FDA decision in sickle cell disease by Dec. 8, and the agency will decide on a second indication in beta thalassemia by March 30, 2024. The drug represents a “multibillion-dollar” opportunity, Vertex Chief Operating Officer Stuart Arbuckle said on the company’s third-quarter earnings call.
The company is “on track” with its launch preparations, which include setting up authorized treatment centers and working with payers. Arbuckle previously noted that about 50 treatment centers in the U.S....
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