BioMarin Pharmaceutical Inc.’s gene therapy Roctavian reduced the annualized rate of bleeding in patients with hemophilia A by 80% in a three-year study.
(Bloomberg) — BioMarin Pharmaceutical Inc.’s gene therapy Roctavian reduced the annualized rate of bleeding in patients with hemophilia A by 80% in a three-year study.
Patients who received the one-time treatment used 94% fewer replacement infusions of factor VIII, a natural clotting substance that’s missing in people with the disorder, BioMarin said Sunday in a statement.
BioMarin recently won European approval of the therapy Roctavian, which treats severe forms of the blood disorder by giving patients a working copy of the faulty gene that causes the inherited disease. BioMarin has said it expects Roctavian’s net price to be about $1.5 million euros ($1.6 million). While that falls within the range a US drug-price review group said would be appropriate, European payers have balked at such high prices for gene therapies before.
The company has signed its first agreement with a German insurance group that will set payments for the drug based on patient outcomes, according to the statement.
Shares of BioMarin have gained about 28% over the past 12 months.
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