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Roche gets wider approval for haemophilia drug Hemlibra

Approved to treat bleeding episodes in patients without inhibitors


The FDA has cleared Roche’s Hemlibra for use in the majority of patients with haemophilia A, extending its use beyond the minority who have developed resistance to factor VIII treatments.

The US regulator has approved the antibody to prevent or reduce the frequency of bleeding episodes in children and adults with haemophilia A without inhibitors, antibodies that bind to factor VIII drugs and stop them working properly.

Hemlibra (eculizumab) was cleared for use in patients with inhibitors last year, and is now the only FDA-approved drug for both sets of patients. It was approved in Europe for patients with inhibitors earlier this year, and is under review at the EMA for a non-inhibitor group with a decision possible before year-end.

The broader indication means that Hemlibra can now challenge the entire range of factor VIII drugs in the marketplace – both short-acting and newer long-acting versions such as Shire’s Adynovate, Biogen’s Eloctate, Bayer’s Kovaltry and CSL Behring’s Afstyla – with the advantage of being dosed every one, two, or four weeks depending on need, rather than multiple times per week.

Hemlibra is a bispecific antibody that binds to factor IXa and factor X, mimicking the role factor VIII plays in the blood, and achieved a significant and clinically meaningful reduction in the number of treated bleeds in clinical trials.

Roche says that Hemlibra is the first drug to show an improvement over standard haemophilia A treatment, cutting bleeds by two-thirds (68%) compared to earlier factor VIII prophylaxis.

“Many preventative treatment options for people with haemophilia A without factor VIII inhibitors require intravenous infusions several times a week,” comments Michael Callaghan, a haematologist at Children’s Hospital of Michigan in the US.

“Even then, people can still experience bleeds, and there has been a need for more treatment options,” he adds, noting that Hemlibra is the first new class of medicine for haemophilia A in almost two decades.

The combination of increased efficacy and easier administration has some analysts predicting stellar sales for Hemlibra, particularly as its cost of around $450,000 per year is actually viewed as providing a saving over the current costs of treating haemophilia A, particularly among patients who go on to develop inhibitors.

Early take-up seems to be strong, with Hemlibra adding $60m to Roche’s revenues in the first half of the year, and analysts are predicting sales above $2b by 2022 and could climb as high as $4bn or $5bn a year at peak.

Article by
Phil Taylor

8th October 2018

From: Regulatory



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