BioMarin Pharmaceutical Inc.’s Roctavian was still effective three years after patients received the one-time treatment for hemophilia A, the company said Sunday while disclosing its first contract with a German insurer for the gene therapy.
(Bloomberg) — BioMarin Pharmaceutical Inc.’s Roctavian was still effective three years after patients received the one-time treatment for hemophilia A, the company said Sunday while disclosing its first contract with a German insurer for the gene therapy.
Roctavian reduced the annualized rate of bleeding in patients with hemophilia A by 80% in a three-year study. The company said 92% of patients weren’t taking prophylactic treatments three years after they received Roctavian. 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 in a statement.
BioMarin will soon submit the data to the US Food and Drug Administration, which requested the follow-up information while it’s reviewing BioMarin’s application for the drug. The additional data may delay the FDA’s decision, currently expected by March 31, by three months. It could be weeks yet before the FDA indicates whether it will need to push the timeline, BioMarin executives said in an interview ahead of the JPMorgan Healthcare Conference.
“We literally got it in Friday,” BioMarin Chief Executive Officer Jean-Jacques Bienaime said of the data. “They know it’s coming, they’re expecting and we’re going to submit it.”
BioMarin recently won European approval of the therapy, 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 ($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. BioMarin will reimburse the insurer should patients relapse in a given period, Chief Commercial Officer Jeff Ajer said in the interview. The company declined to share specifics on the contract for “competitive reasons.”
BioMarin anticipates it will announce similar contracts with the other two large German insurers in a matter of “weeks,” Ajer said. The company’s already talking to US insurers ahead of the FDA’s decision. “Several” large payers have approached BioMarin about their interest in covering the gene therapy, Ajer said, which BioMarin sees as a “good signal.”
In the US, the company plans to offer a warranty on the gene therapy to all insurers so it won’t need to negotiate with each payer, he said.
Shares of BioMarin have gained about 28% over the past 12 months.
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