Novo Nordisk's Alhemo Wins FDA Nod, Expanding Subcutaneous Option For Hemophilia Patients

The U.S. Food and Drug Administration approved on Friday, the Novo Nordisk A/S (NYSE:NVO) Alhemo (concizumab-mtci) injection as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in patients 12 years of age and older with hemophilia A or B without inhibitors.

In December 2024, the FDA approved Alhemo for patients 12 years of age and older with hemophilia A or B with inhibitors.

Currently, many hemophilia A or B treatments without inhibitors are administered via intravenous infusions. With this approval, Alhemo now offers a subcutaneous injection treatment option for this population.

Alhemo is designed to block a protein called tissue factor pathway inhibitor (TFPI), which stops blood from clotting.

Also Read: Pfizer Seeks Label Expansion For Hemophilia Drug After Promising Data

By inhibiting TFPI, Alhemo improves the production of thrombin, which helps to clot the blood and prevent bleeding, when clotting factors VIII and IX are missing or deficient, regardless of inhibitor status.

The primary objective of the pivotal phase 3 explorer8 trial was to compare the number of treated spontaneous and traumatic bleeding episodes, as measured by the ABR, in patients aged 12 years and older with HA/HB without inhibitors, receiving Alhemo prophylaxis versus no prophylaxis (on demand Factor VIII/Factor IX treatment).

The study showed a statistically significant reduction in ABR of 79% for hemophilia B patients and 86% for hemophilia A patients.

Additionally, notable reductions in the average and median ABRs further reinforced the efficacy of Alhemo prophylaxis in reducing bleeds across both patient groups, with an average ABR of 3.1 and a median ABR of 1.6 for hemophilia B patients, compared to 14.8 and 14.9, respectively, and an average ABR of 2.7 and median ABR of 2.9 for hemophilia A patients, compared to 19.3 and 19.6, respectively.

Alhemo is provided in prefilled, premixed pens for subcutaneous injection (60 mg/1.5 mL, 150 mg/1.5 mL, or 300 mg/3 mL) via thin 32 gauge, 4 mm needles, which are provided separately, offering an additional treatment choice for patients with hemophilia with inhibitors, and now without inhibitors.

In February, Novo Nordisk reported interim results from the phase 3 FRONTIER3 trial of 70 children (aged one-11 years old) with hemophilia A with and without inhibitors.

The median ABR was zero; 74.3% of participants had zero treated bleeds.

All children with hemophilia A with inhibitors (n=14) reported zero treated bleeds. The data showed that Mim8 was well-tolerated and efficacious.

The company expects Mim8 regulatory submission during 2025.

In March this year, the FDA approved Sanofi SA's (NASDAQ:SNY) Qfitlia (fitusiran), the first antithrombin-lowering (AT) therapy for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A or B with or without factor VIII or IX inhibitors.

NVO Price Action: NVO stock is up 1.91% at $47.97 at publication on Friday.

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