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US drug pricing watchdog will probe BioMarin's hemophilia gene therapy

U.S. pricing watchdog the Institute for Clinical and Economic Review said that it will investigate BioMarin Pharmaceutical Inc.'s hemophilia A gene therapy valoctocogene roxaparvovec for cost-effectiveness.

ICER said that likely comparators for the cost assessment will be currently approved treatments for hemophilia A like factor VIII replacement therapy and Hemlibra, a drug from Roche Holding AG's Genentech. BioMarin's gene therapy is not yet approved, but the U.S. Food and Drug Administration is expected to make a decision by the middle of the year.

People with hemophilia A are missing or have low levels of a protein called factor VIII, and valoctocogene roxaparvovec introduces a gene in a single infusion that would enable the production of the protein.

BioMarin submitted its biologics license application to the FDA in December 2019, and the European Medicines Agency accepted the application in November the same year. Both regulators have designated it an orphan therapy with the U.S. additionally calling it a breakthrough.

ICER proposed changes to its cost effectiveness framework for gene therapies that could potentially be one-time "cures" for diseases rather than drugs that treat chronic conditions. Gene therapies like Novartis AG's Zolgensma or Spark Therapeutics Inc.'s Luxturna prompted pricing concern with initial pricetags of more than $1 million but a high potential for benefit for patients with rare diseases.

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