The headquarters of US biopharmaceutical company Vertex Pharmaceuticals in Boston, Massachusets, on November 4, 2023. The U.S. health regulator has approved Vertex Pharmaceuticals and CRISPR Therapeutics' gene therapy to treat a rare blood disorder requiring regular blood transfusions, in patients 12 years and older, Vertex said on Tuesday. The decision earns the therapy, branded as Casgevy, the second U.S. approval after it was greenlighted in December for sickle cell disease, another inherited blood disorder. The approval by the Food and Drug Administration comes more than two months ahead of its expected action date of March 30. Oppenheimer analyst Hartaj Singh said he expects a "slow and steady launch" for the therapy and estimated combined peak sales of about $400 million.

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Persons: Oppenheimer, Hartaj Singh Organizations: Vertex Pharmaceuticals, Pharmaceuticals, CRISPR Therapeutics, Food and Drug Administration Locations: US, Boston, Massachusets, U.S, United States