Committee recommends approval in EU of concizumab for hemophilia
Developer seeking therapy's approval for hemophilia A and B with inhibitors
A European Medicines Agency (EMA) committee has recommended the approval of Novo Nordisk’s concizumab as a prophylactic, or preventive, treatment for adults and children, ages 12 and older, with hemophilia A or B who have inhibitors.
The recommendation, from the EMA’s Committee for Medicinal Products for Human Use (CHMP), will now be reviewed by the European Commission, which has final say over therapy approvals in the European Union.
While the commission is not required to abide by the CHMP’s recommendations, it almost always does. If approved, the therapy will be sold under the brand name Alhemo.
“The positive opinion from the CHMP for Alhemo is a major milestone for patients,” Stephanie Seremetis, MD, chief medical officer for hemophilia at Novo Nordisk, said in a company press release.
A final decision is expected in about two months, according to Novo Nordisk. The company noted that, if ultimately approved, concizumab would be the first preventive treatment in Europe offering once-daily, under-the-skin (subcutaneous) dosing for this patient population.
“If approved, Alhemo would be an important addition to our growing [hemophilia] portfolio as it offers the potential of everyday prophylaxis to prevent bleeds for people living with [hemophilia] who have developed the complication of inhibitors,” Seremetis said.
Concizumab approved in Canada, Japan, under brand name Alhemo
The therapy was approved in Canada last year, under the brand name Alhemo, as a treatment for both hemophilia A and hemophilia B in patients ages 12 and older with inhibitors. It is also cleared for use in Australia, Sweden, and Japan under the same brand name.
However, an application for concizumab’s approval in the U.S. was rejected by the U.S. Food and Drug Administration early last year, with requests for more information on the monitoring of patient dosing and the treatment’s manufacturing process. Novo Nordisk has already refiled its application seeking the therapy’s approval in the U.S., according to an earnings call hosted by the company in July.
Hemophilia comprises a group of disorders characterized by excessive bleeding due to deficiencies in the proteins that normally help the blood to form clots. Hemophilia A is marked by a deficiency of the clotting protein factor VIII, known as FVIII, while hemophilia B is marked by a deficiency of clotting factor IX, or FIX.
Standard treatment for both types of hemophilia involves replacement therapy, in which a version of the deficient clotting factor is administered to patients. This is effective for managing bleeds for many individuals, but in some cases, the patient’s immune system mistakenly recognizes the replacement therapy as a threat.
When this happens, immune cells make antibodies, commonly called inhibitors, against the therapy, which can limit its effects.
Concizumab works by blocking the activity of a protein called TFPI, which normally helps to prevent unneeded blood clotting. By suppressing TFPI, the medication is designed to promote blood clotting irrespective of the patient’s hemophilia type or inhibitor status.
This [therapy] could enable patients to have greater confidence in the pursuit of daily activities, which is especially important for people living with hemophilia B with inhibitors, who currently have very limited treatment options.
The therapy is available in a portable, premixed and prefilled pen, and it is administered via once-daily subcutaneous injection.
According to Seremits, concizumab “could alleviate the physical, emotional, and overall treatment burden for people living with hemophilia, as it is delivered in a pre-filled, multi-use, portable pen that can be stored at room temperature for up to four weeks.”
As such, treatment administration is expected to be more convenient for patients.
“This [therapy] could enable patients to have greater confidence in the pursuit of daily activities, which is especially important for people living with hemophilia B with inhibitors, who currently have very limited treatment options,” Seremitis said.
Alhemo’s approvals, as well as the recent CHMP recommendation, were based mainly on data from a Phase 3 clinical trial called explorer7 (NCT04083781), which tested concizumab in hemophilia A and B patients with inhibitors. The study’s results showed the therapy reduced bleeding rates by more than 85%.
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