FDA, Regulatory

Unencumbered by safety concerns, Roche’s Hemlibra gets breakthrough status for new patient group

Recent safety concerns have not stopped Roche from obtaining a speedy FDA review for using Hemlibra to treat hemophilia A patients without factor VIII inhibitors, a market segment crucial to Roche’s blockbuster quest for its new therapy approved last November.

Sandra Horning

Roche rattled the market when it reported five deaths among patients taking Hemlibra, including two that emerged earlier this year (The company emphasized that all five were unrelated to the drug). Jefferies analyst David Steinberg pointed out that heightened concerns could impact use in “non-inhibitor patients who are generally well controlled” on Advate, a factor replacement therapy from chief rival Shire.

But the FDA handed over a breakthrough therapy designation, evidently happy with data from the Phase III Haven 3 study during which Roche says no thrombotic microangiopathy or thrombotic events occurred. Forming the basis of the designation, the trial showed that Hemlibra prophylaxis dosed subcutaneously had a statistically significant reduction in treated bleeds. According to Roche, it was also superior to prior factor VIII treatment when comparing the effects of the two in the same patient.

“Hemlibra is the first medicine to show superior efficacy compared to factor VIII prophylaxis, the standard of care for people with haemophilia A without inhibitors, in an intra-patient comparison,” said Sandra Horning, Roche’s CMO, in a statement. “We look forward to working with health authorities to make Hemlibra available to people without inhibitors as soon as possible.”

Pressure is mounting on Shire, the rare disease player for whom hemophilia is a key franchise. Hemlibra is approved in both the EU and Japan. With the FDA now agreeing to expedite the development and review of its expanded use, the threat is only going to grow bigger.


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