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.
uniQure N.V./CSL Limited’s Hemgenix (tranacogene dezaparvovec-drlb) has been approved for the treatment of adults with moderately severe to severe hemophilia B by the US Food and Drug Administration, making it the first gene therapy for the rare bleeding disorder and the most expensive drug to date.
The Australian firm revealed the treatment would cost $3.5m in the US, edging past the $3m price tag for
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Sun’s Leqselvi goes up against Lilly's Olumiant and Pfizer's Litfulo, which currently has the broadest label, in the US alopecia market. Can it shake things up early, amid growing activity in the space with several new mechanisms of action being investigated?
Nanoscope’s experimental gene therapy, MCO-010, is designed to target the broader retinitis pigmentosa population, regardless of the underlying gene mutation, with a completed US regulatory submission expected in early 2026.
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
The approval for young children, consistent with the FDA’s new COVID-19 vaccine policy, restricts Spikevax to those with conditions putting them at higher risk.
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Big pharma is increasingly looking east to restock its pipelines, a process that is helping to forge a new generation of mid-sized R&D-based companies in China and is reflected in their stock price performance.
Nanoscope’s experimental gene therapy, MCO-010, is designed to target the broader retinitis pigmentosa population, regardless of the underlying gene mutation, with a completed US regulatory submission expected in early 2026.
