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In blow to Shire, Roche bags an FDA OK for its blockbuster hemophilia A prospect

Guy Young, USC Keck School of Medicine. Image: USC

Despite being dogged by questions about safety and an unusual legal counterattack from rival Shire, Roche today scored an FDA approval for emicizumab (ACE910), its hemophilia A therapy which will now hit the market as Hemlibra.

The drug is designed to bring together factor IXa and factor X proteins to restore the blood clotting process for hemophilia A patients, reducing the threat of bleeds. And analysts have pegged potential sales at up to $5 billion, though Reuters‘ most recent consensus was a considerably less frothy $1.6 billion.

Significantly, the approval comes more than three months ahead of its late-February PDUFA date, registering as the 40th new molecular entity of the year as the industry looks ready to cruise past the current-era record 44 new approvals achieved at CDER in 2015.

The approval was based on some impressive data on a drug that Roche has steadfastly maintained is a near certain blockbuster-to-be. But it also comes with a boxed warning on a threat of blood clots and damage to blood vessels, which could also crimp sales.

Last summer the pharma giant laid out data detailing how the drug reduced the rate of treated bleeds by 87%, hitting the primary endpoint, the drug also handily outscored bypassing agents like Shire’s. Their therapy slashed “on-demand BPAs across all bleed measurements, including zero treated bleeds (62.9 percent vs. 5.6 percent), zero treated spontaneous bleeds (68.6 percent vs. 11.1 percent), zero treated joint bleeds (85.7 percent vs. 50.0 percent), zero treated target joint bleeds (94.3 percent vs. 50.0 percent) and zero bleeds overall, which includes all treated and non-treated bleeds (37.1 percent vs. 5.6 percent).”

Along the way, Roche blamed Shire’s bypassing agents for thromboembolic events that they saw in their study, advising doctors to stop using FEIBA.

Shire, to put it mildly, flipped.

“To imply a cause-and-effect of FEIBA having caused the severe adverse events is misleading,” Shire hematology chief Juliana Dierks told Reuters. “We are looking forward to transparency. Give us the data, give us the facts.”

Shire also claimed that Roche has been misleadingly offering a look at “treated bleeds” — a secondary endpoint — instead of the “number of bleeds over time,” originally laid out as the primary endpoint in HAVEN 1. And by blaming thromboembolic incidents in the study — with one patient dying of a rectal hemorrhage — on its bypassing agent, Shire also maintained that it had been damaged. Shire sought an injunction against Roche in Germany, but the action was rejected by the court and the complaint hasn’t slowed its Big Pharma rival at all.

How bad could this get for Shire? Bernstein analysts say Hemlibra along with new therapies from Novo Nordisk and Bayer could shrink its share of the hemophilia market from 49% to 29%

Roche has never done anything but shrug the assault off. The pharma giant is losing patent protection on three franchise drugs, and it needs to field major new drugs to fill the gap.

Hemlibra is priced to do just that. Roche, like a growing number of companies, is using an average wholesale price based on average weight. At an average weight of about 127 pounds, Roche says, the cost “is approximately $482,000 for the first year of treatment and then approximately $448,000 per year, which is less than half of the WAC of the only other approved prophylactic treatment for these patients.”

The price fits in at the lower rung of the list of the 10 most expensive drugs in the world, all among the rare disease crowd.

“Today’s approval provides a new preventative treatment that has been shown to significantly reduce the number of bleeding episodes in patients with hemophilia A with Factor VIII inhibitors,” said Richard Pazdur, whose titles include acting director of the Office of Hematology and Oncology Products. “In addition, patients treated with Hemlibra reported an improvement in their physical functioning.”

“People with hemophilia A who develop inhibitors face significant challenges preventing bleeds and typically require infusions of medicine multiple times a week, which can be especially difficult for young children and their families,” said Guy Young, director of hemostasis and thrombosis program, Children’s Hospital Los Angeles, and professor of pediatrics, University of Southern California Keck School of Medicine. “This new medicine has been shown to reduce the frequency of bleeds compared to the currently available medicines and only needs to be injected once a week. This could make a meaningful difference for these children.”

Image: Roche, John McCarter

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
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Atlas Venture Cambridge, MA

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