Roche poised for push into haemophilia market

Roche is aiming to shore up its place in the haemophilia drug market, with Hemlibra (emicizumab) on the brink of regulatory approval for expanded use.

The US Food and Drug Administration (FDA) is this week expected to grant the Swiss pharma giant approval so the drug can be used to treat haemophilia A.

This follows the results of a phase 3 Haven 3 trial, which showed that Hemlibra works in patients aged 12 years or older who have not produced inhibitors, unlocking a large portion of the market in patients treated with standard care of recombinant factor VIII therapy.

The drug is already approved for patients for whom factor VIII replacement therapy does not work due to their bodies producing inhibitors.

Factor VIII is essential in the blood clotting process but if it is absent due to defects in the X-linked F8 gene, an individual will develop haemophilia A, which can be fatal.

The market for haemophilia is worth £7.7 billion so Roche is understandably keen to secure approval. The decision is expected to be announced by tomorrow [October 4].

A report by Reuters states: “To hit $5 billion global annual sales that some analysts forecast, Hemlibra must wrest business away from haemophilia giants like Shire, Bayer, Novo Nordisk and Octapharma, whose ‘clotting factors’ are now standard for people who lack proteins that stop bleeding.”

It went on to say that these rivals build up customer loyalty by providing free events and summer camps for sufferers and their families to maintain their position. Shire, Bayer and Octapharma did not respond to requests for comment on their efforts to boost ties to patients, said Reuters.

Roche is said to be “convinced [that] word of mouth in the haemophilia community, backed up by its studies showing that Hemlibra shots may be more effective and must be administered less frequently than infusions of clotting factors, will overcome any current allegiances.”

Roche, which is known for its cancer drugs, such as Rituxan, Avastin, and Herceptin, is looking for new revenue sources such as rare disease drugs, as its existing patents expire and cheaper biosimilars enter the market.

The FDA has yet not issued any comment on the approval of Hemlibra.