Pfizer’s Gene Therapy Asset Chases UniQure/CSL’s Hemgenix After Pivotal Hemophilia B Success
Pfizer’s fidanacogene elaparvovec has hit the mark in a Phase III hemophilia B trial just a month after rival UniQure’s Hemgenix won US approval, with Pfizer’s plans for regulatory action raising the stakes for Hemgenix.
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While Hemgenix has courted criticism since its $3.5m price tag was unveiled, a drug pricing expert has told Scrip discounts and value-based agreements could make the therapy more palatable to payers.
The firms’ gene therapy Hemgenix has won a US thumbs up for the treatment of hemophilia B and plans are underway for a US launch with a big price tag that experts agree should pay off in the long-term.
Pfizer expands its position in rare diseases in a collaboration with Spark Therapeutics and also licenses a pair of preclinical oncology candidates from Belgium’s iTeos Therapeutics. Meanwhile, Shire gets $15 million in research funding from the Cystic Fibrosis Foundation to help advance a novel aerosolized therapy addressing the underlying cause of CF.