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Roche gets first approval for haemophilia A therapy

The first-in-class therapy wins US marketing licence three months earlier than expected

Roche

Roche has bagged an early FDA approval for haemophilia A drug Hemlibra, after a development process dogged by safety issues and a legal spat with Shire.

Hemlibra (emicizumab) has been approved to prevent or reduce the frequency of bleeding episodes in patients with haemophilia A who have developed inhibitors to Factor VIII, according to the FDA, which said in a statement that the new drug has been shown to reduce the number of bleeding episodes in these patients as well as improve physical functioning.

Roche’s drug is a first-in-class therapy - delivered by subcutaneous injection once a week - that works by bridging other factors in the blood to restore blood clotting, said the agency. It’s been approved around three months earlier than expected.

With Hemlibra Roche is trying to break into a market for haemophilia drugs worth around $7bn in the world's top seven markets (US, France, Germany, Italy, Spain, the UK and Japan) last year. The market for haemophilia A with inhibitors is currently dominated by Shire and Novo Nordisk with their Feiba and NovoSeven bypassing agents (PBAs).

Roche’s chief medical officer Sandra Horning said that the approval “represents an important advancement for people with haemophilia A with inhibitors, who have struggled to manage their bleeding disorder and haven’t had a new medicine in nearly 20 years”.

In the HAVEN 1 trial, Hemlibra achieved an 87% reduction in the rate of treated bleeds - cutting them from an average of 23.3 per year in patients with no prophylactic treatment to 2.9. A second study - HAVEN 2 - showed that the drug was able to completely do away with bleeding episodes in 87% of patients.

The data was well-received by haematologists and led to speculation that Roche had a potential blockbuster on its hands, with peak sales predictions in the region of $1.5bn a year.

Roche has said it will press ahead with a US launch shortly, and intends to price the drug at $482,000 in the first year of treatment, dropping to $448,000 in subsequent years, which it claims is less than half the wholesale acquisition cost of its rival. Its hoping for approvals in Europe and Japan next year.

One issue the company will have to contend with however is a black box warning on Hemlibra’s label that the drug has been linked to severe blood clots that can damage blood vessels in patients who were also given a rescue treatment (activated prothrombin complex concentrate) to treat bleeds.

Those side effects have been a bone of contention with Shire launching a lawsuit against its rival over what it claimed were misleading statements - but despite getting a preliminary injunction was ultimately unsuccessful.

Shire took issue with Roche's assertion that the events occurred when Feiba was used concurrently with emicizumab, saying this “unlawfully disparaged Shire's proven bypassing agent”. Shire's inhibitor therapies collectively made sales of $452m last year.

Article by
Phil Taylor

17th November 2017

From: Regulatory

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