US Approves Novo Nordisk's Daily Hemophilia Drug Alhemo

Novo Nordisk has finally secured approval from the US Food and Drug Administration for its new hemophilia medication, Alhemo, following an earlier rejection by the agency last year. This once-daily injectable treatment is now authorized to decrease or prevent bleeding episodes in individuals aged 12 and older who have hemophilia A or B with inhibitors. The company has yet to disclose the US pricing for Alhemo.

This approval marks a positive close to the year for the Danish pharmaceutical giant, even though it doesn't completely counterbalance the negative impact from the disappointing results of its CagriSema study revealed earlier. Despite last Friday's setback, Novo Nordisk shares rebounded by about 5% in early trading on the following Monday, recovering from a nearly 20% loss triggered by the release of the CagriSema data.

Alhemo, which was previously known under the name concizumab, is an anti-tissue factor pathway inhibitor (TFPI). By obstructing TFPI, Alhemo effectively increases thrombin levels. Thrombin is crucial for blood clot formation in patients who have developed inhibitors, which are antibodies that can neutralize clotting factors like factor VIII or IX. In pivotal trials, Alhemo demonstrated a significant reduction in the frequency of treated spontaneous and traumatic bleeds by 86% compared to patients without preventive care.

The new treatment from Novo Nordisk is administered subcutaneously via a pen device and is available in three different dosage strengths. Novo claims that Alhemo is the first subcutaneous treatment of its type for this patient group, contrasting with other treatments that are primarily intravenous.

Meanwhile, Pfizer has also entered the hemophilia market with its product, Hympavzi, which was approved in the US this past October. Like Alhemo, Hympavzi functions by inhibiting the TFPI mechanism and is delivered subcutaneously using a pen injector, though its administration is weekly instead of daily. However, Hympavzi is only approved for hemophilia A and B patients without inhibitors.

Novo Nordisk estimates that approximately 30% of individuals with severe hemophilia A and 5-10% of those with severe hemophilia B will develop inhibitors. This demographic potentially broadens Alhemo's reach compared to Pfizer's Hympavzi. However, Pfizer is preparing to compete vigorously; data on Hympavzi in patients with inhibitors is expected by 2025, which could lead to a label expansion, thereby matching Alhemo's regulatory status but with a more convenient dosing schedule.

The market leader in this space is Roche's Hemlibra, a weekly injection that generated sales around $4.6 billion last year. Hemlibra is approved for hemophilia A with or without inhibitors, but it does not treat the B type of the disease. Novo Nordisk is positioning Alhemo to compete in this segment. Additionally, Novo is working on another promising candidate, Mim8, which successfully passed its Phase 3 trials in May. Mim8 aims to challenge Hemlibra by offering the convenience of a once-monthly dosing schedule, potentially giving Novo a competitive edge.