Alhemo Once-Daily Treatment for Hemophilia A, B with Inhibitors FDA Approved

The FDA recently approved Novo Nordisk’s Alhemo (concizumab-mtci), the first subcutaneous, once-daily treatment for hemophilia A or B with inhibitors, designed to prevent or reduce bleeding episodes in patients aged 12 and older.

The drug targets the tissue factor pathway inhibitor (TFPI), which can prevent blood from clotting properly, according to a release by Novo Nordisk. By blocking TFPI, Alhemo increases the production of thrombin, a protein essential for blood clotting, which reduces the frequency of bleeding episodes in patients with inhibitors.

This treatment addresses a critical need for those with hemophilia.

For instance, 30% of patients with severe hemophilia A and 5% to 10% of those with severe hemophilia B develop inhibitors, which complicates their condition. For those with hemophilia B and inhibitors, treatment options are especially limited.

The FDA granted Breakthrough Therapy designation to Alhemo based on results from Phase 2 clinical trials.

Anna Windle, senior vice president of Clinical Development at Novo Nordisk, said, “The approval of Alhemo signifies a remarkable achievement in prophylactic hemophilia treatment for individuals with inhibitors aged 12 years and older who, in some cases, currently have few options.”

Many current treatments for hemophilia A or B with inhibitors require intravenous infusions, which can be difficult and time-consuming.

Alhemo uses a prefilled, premixed pen for subcutaneous injection with a thin 32-gauge, 4-mm needle. This makes the treatment process easier and less invasive.

Hemophilia is a rare genetic disorder that affects the body’s ability to form blood clots due to a lack of clotting factors. This condition can cause prolonged bleeding after injuries, surgeries or random bleeding episodes in muscles, joints and organs, which can become life-threatening if not managed effectively.

Inhibitors can add another layer of challenges to treating hemophilia. These are antibodies that block the activity of clotting factors, making traditional therapies less effective.

In the U.S., around 33,000 males live with hemophilia, with Hemophilia A making up 80% to 85% of cases. Approximately 30% of patients with hemophilia A and 5-15% of those with hemophilia B develop inhibitors.

Clinical trials have shown Alhemo to be both effective and safe. For example, in a Phase 3 trial with 133 male patients with hemophilia A or B with inhibitors, researchers compared bleeding episodes between patients using Alhemo and those without prophylaxis.

The results showed an 86% reduction in the annualized bleeding rate for those on Alhemo.

Common side effects reported in clinical trials include injection site reactions and hives, skin redness, rash, itching and abdominal pain. It’s suggested that patients with a history of severe allergic reactions to Alhemo or its ingredients should avoid using it. The treatment also carries a risk of blood clots, so careful monitoring is critical.

While the cost of the drug has not yet been fully disclosed, the average yearly cost of hemophilia medication can exceed $393,000 per patient. Hemophilia treatment costs vary depending on factors such as how often a patient experiences bleeding episodes and how much treatment they need for each episode, according to Evernorth Health Services.

Alhemo offers a new way to manage hemophilia A or B with inhibitors. It makes treatment more efficient and helps solve some of the challenges that come with the condition, giving patients more options for their care.