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FDA Approves Hympavzi For Hemophilia

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New Hemophilia Drug Helps Prevent Bleeding Episodes

Oct. 14, 2024 – The FDA has approved a new preventive treatment for people with certain types of hemophilia that can reduce bleeding episodes, sometimes down to just a few per year.

Hemophilia is a rare blood clotting disorder that can lead to prolonged bleeding after an injury or surgery, as well as spontaneous bleeding in the muscles, joints, or organs. The new treatment, called Hympavzi, is for people ages 12 and older with hemophilia A or hemophilia B. Those are the two most common types of the condition and differ based on specific proteins involved in blood clotting.

The medicine is a once-per-week shot using a prefilled pen and offers an alternative to current preventive options that involve infusions several times per week. Its generic name is marstacimab. 

"Today's approval of Hympavzi provides patients with hemophilia a new treatment option that is the first of its kind to work by targeting a protein in the blood clotting process," Ann Farrell, MD, director of the Division of Non-Malignant Hematology in the FDA's Center for Drug Evaluation and Research, said in a statement. "This new type of treatment underscores the FDA's commitment to advance the development of innovative, safe and effective therapies."

Rather than helping the blood clot normally by adding clotting ability through infusions, Hympavzi works by reducing the amount of a naturally occurring protein that thins the blood. The body then has more of an enzyme called thrombin that is important for clotting, which ultimately leads to fewer bleeding episodes.

The approval is based on results of a clinical trial involving 116 men and boys, all of whom had hemophilia A or hemophilia B with severe symptoms, meaning they bleed after an injury and regularly have spontaneous bleeding without an obvious cause. For six months, 33 of them used standard on-demand treatments when they bled, and the other 83 used standard preventive treatments. Then, for the next 12 months, all of the people in the study took Hympavzi preventively.

The researchers compared the estimated annual number of treated bleeding episodes, during the six-month standard treatment period, to the annual rate when the people in the study took Hympavzi. On-demand standard treatment averaged the equivalent of 38 episodes per year, compared to 3.2 per year with Hympavzi, the FDA reported in its approval announcement.

Preventive standard treatments averaged the equivalent of 7.85 episodes per year, compared to 5.08 per year with Hympavzi. The FDA called that rate with Hympavzi "similar" to the rate with standard preventive treatments used in the study.

In a news release, drugmaker Pfizer said Hympavzi reduced the annual bleeding rate for treated bleeds by 35% among the group that initially took standard preventive treatments, and by 92% among the group that initially took on-demand treatments.

Side effects while using Hympavzi may include reactions where the needle goes in, headache, and itching. The FDA also noted that people using Hympavzi need to consider warnings and precautions about circulating blood clots (also called thromboembolic events), hypersensitivity, and potential toxic effects on a developing embryo or fetus.

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%.

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