Novo Nordisk closes on EU okay for haemophilia antibody

Novo Nordisk's anti-TFPI antibody concizumab has been backed for approval in the EU as a once-daily drug to prevent bleeding episodes in people with haemophilia.

The recommendation by the EMA's human medicines committee, the CHMP, sets up the drug to become the first once-daily subcutaneous prophylactic treatment for people aged 12 years or older living with haemophilia A or B with inhibitors in the EU.

Concizumab – which has the trade name Alhemo – is already approved in other markets, including Japan, Australia, and Switzerland, but was rejected by the FDA last year with a request for more information related to the monitoring and dosing of patients.

It is one of an emerging class of therapies that block TFPI (tissue factor pathway inhibitor), a protein which prevents blood from clotting.

Just last week, Pfizer's Hympavzi (marstacimab) became the first drug in the anti-TFPI class to be approved in the US and was also recommended for approval by the CHMP in September, with an approval expected in the next few weeks.

However, the two drugs are going after different indications. Alhemo is indicated for use in haemophilia A and B patients with inhibitors – antibodies stimulated as an immune response to clotting factor replacement therapy – while once-weekly injectable Hympavzi is intended to treat patients without inhibitors.

Both drugs will compete in haemophilia A with Roche's hugely successful Hemlibra (emicizumab), which can be dosed as infrequently as four weeks in some patients and is approved for patients both with and without inhibitors.

The drug – which has been on the market since 2017 – achieved sales of CHF 2.1 billion ($2.43 billion) in the first half of this year, with growth being driven by use in the non-inhibitor patient population, according to Roche's second-quarter update.

Novo Nordisk said in a statement that up to 30% of people living with severe haemophilia A develop inhibitors, while for severe haemophilia B the rate is 5% to 10%.

The company's chief medical officer for haemophilia, Stephanie Seremetis, said approval by the EMA will be a "major milestone for patients" as it "offers the potential of everyday prophylaxis to prevent bleeds for people living with haemophilia who have developed the complication of inhibitors."

She added that it could "alleviate the physical, emotional, and overall treatment burden for people living with haemophilia, as it is delivered in a pre-filled, multi-use, portable pen that can be stored at room temperature for up to four weeks."

In contrast, Hemlibra is stored in vials in the refrigerator and requires a regular syringe to administer. Hympavzi, meanwhile, is available in a pre-filled syringe as well as a pen format but also needs to be refrigerated, although its label says it is okay to store one time only at room temperature for up to seven days.

The CHMP's decision is based on the results of the Explorer7 trial, which showed that concizumab reduced the number of treated bleeds in patients by 86%.