UPDATE 3-U.S. FDA expands approval of Roche hemophilia drug

Roche on Thursday said U.S. regulators approved expanded use of its drug Hemlibra to include almost all patients with haemophilia A, as the Swiss drugmaker increases its focus on diseases beyond cancer to help replace revenue from older products that have lost patent protection.

The Food and Drug Administration cleared Hemlibra to treat adults, children and newborns with the genetic disorder in which blood does not clot properly, putting them at risk of dangerous bleeding.

Hemlibra can now be used for patients who have not become resistant to standard treatment, known as factor replacement therapy. It is the only preventive treatment for haemophilia A patients with and without factor VIII inhibitors that can be self-administered subcutaneously once weekly, every two weeks or every four weeks. Older treatments, sold by companies like Shire Plc, require more frequent intravenous infusions.

Hemlibra won initial U.S. approval last year only for haemophilia A patients with factor VIII resistance or inhibitors. That accounted for only about 5 per cent to 7 per cent of the estimated 16,000 U.S. haemophilia A patients. Factor VIII is a protein involved in clotting.

For a haemophilia A patient of the average weight of about 140 pounds (63.4 kg), the annual list price for Hemlibra will be about $492,000 per year, following the initial first-year dose that costs an additional $38,000, a spokeswoman for Roche's U.S. Genentech unit said on Thursday. Dosing is based on a patient's weight.

Roche is looking to capture a big share of a $10 billion-per-year haemophilia market from established rivals like Shire, Bayer AG and Novo Nordisk A/S, which sell clotting factor drugs that have been the standard treatment for decades.

Some analysts have forecast eventual Hemlibra annual sales reaching as high as $5 billion.

Roche is counting on new medicines like Hemlibra to replace falling revenue from its trio of blockbuster cancer medicines, Rituxan, Avastin and Herceptin, which account for about $21 billion in annual sales but will face increasing competition from less expensive biosimilar alternatives.

"I'm hopeful (the expanded approval) will greatly increase the number of people receiving prophylactic treatment, because one of the major reasons that some people don't do prophylaxis is it's just too burdensome, too challenging to do," Dr. Gallia Levy, head of Roche's Hemlibra program, told Reuters in an interview prior to the FDA approval.

"Those people that are not doing prophylaxis, they're bleeding a fair amount. The average is about 30 bleeds a year," Levy said.

Haemophilia affects only about 20,000 people in the United States and roughly 400,000 around the world. But the high price of treatment has made it a very attractive area for drug company research.

Hemophilia A is the most common form, with about four times as many people with that condition as with haemophilia B.

(With inputs from agencies.)