Just before the end of 2024, Novo Nordisk nabbed US Food and Drug Administration (FDA) approval for its awaited hemophilia drug Alhemo (concizumab-mtci).
Alhemo is approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors.
Patients with hemophilia complicated by inhibitors are a group that has historically faced limited options and significant health risks.
Hemophilia is a rare, inherited bleeding disorder in which the blood does not clot properly due to a deficiency in clotting factors. It affects an estimated 800,000 people globally and 32,000 in the US.
Hemophilia A is characterized by a deficiency in factor VIII, while hemophilia B involves a deficiency in factor IX. Standard treatment typically involves replacement therapy, where patients receive infusions of the missing clotting factor.
However, some patients develop inhibitors — antibodies that neutralize the efficacy of replacement therapies. These inhibitors make managing bleeding episodes more challenging, increasing the risk of severe complications such as joint damage, chronic pain and life-threatening hemorrhages.
Alhemo is a tissue factor pathway inhibitor (TFPI) antagonist designed to prevent or significantly reduce the frequency of bleeding episodes.
The drug is administered subcutaneously, providing a more convenient alternative to traditional intravenous infusions required for factor replacement therapy in patients with hemophilia A or B with inhibitors. The prophylactic comes in prefilled, premixed pens for injection.
In a press release announcing the approval, Novo said that Alhemo is the first subcutaneous injection of its kind in this patient population.
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Approximately 30 percent of patients with severe hemophilia A and five to ten percent of those with severe hemophilia B develop inhibitors, significantly complicating treatment.
Despite advances in hemophilia care, patients with hemophilia B and inhibitors continue to face substantial disease and treatment burdens due to limited prophylactic options for preventing bleeding.
Alhemo works by inhibiting TFPI, which enhances factor Xa (FXa) production during the initiation phase of coagulation. This, in turn, leads to improved thrombin generation — a crucial protein involved in blood clotting. This helps to prevent bleeding when other clotting factors are absent or deficient due to the presence of inhibitors.
Alhemo is a bispecific monoclonal antibody that mimics the function of factor VIII without requiring direct replacement of the missing factor. This mechanism bypasses the inhibitors, allowing the body to achieve clotting even in the presence of factor-neutralizing antibodies.
The FDA approval of the treatment was supported by data from Novo’s late-stage explorer7 study, which found that Alhemo helped reduce annual bleeding rate (ABR) by 86 percent compared to patients who did not receive prophylaxis.
On a key secondary efficacy endpoint, 64 percent of patients receiving Alhemo had no treated spontaneous or traumatic bleeds during the initial 24 weeks of treatment, compared to only 11 percent of patients who did not receive prophylaxis during the same period.
“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,” said Anna Windle, PhD, SVP Clinical Development, Novo Nordisk, in the company’s news release.
“As the first treatment of its kind for this population, Alhemo represents a significant step in helping to address the unmet needs of patients with hemophilia with inhibitors, highlighting Novo Nordisk’s commitment to patient-centric innovations in rare diseases.”
Related: Hympavzi (Marstacimab): FDA Approves First Once-Weekly and Pfizer’s Second Hemophilia Therapy
Although Novo has finally secured regulatory approval for the drug in the US, the journey was a bit rocky.
In 2020, the FDA ordered Novo to pause clinical trials of the drug following reports of blood clots in three patients. After a five-month suspension, Novo resumed the studies.
However, in May of the following year, Novo executives disclosed during an earnings call that the FDA had issued a complete response letter requesting additional details on both dosing and the manufacturing process for concizumab.
Novo secured the approval just after Pfizer received the FDA nod for its TFPI drug Hympavzi (marstacimab-hncq) in October 2024.
However, Hympavzi is indicated for hemophilia A or B patients who have not developed antibodies to prior inhibitor treatments. Hence the two drugs target different patient populations and are not likely to be direct competitors. Hympavzi is administered once weekly.
On the other hand, Novo is developing Mim8, a bispecific antibody that works to bridge Factor IXa and X to replace missing Factor VIII. It would rival Roche’s hemophilia blockbuster Hemlibra (emicizumab-kxwh), which is indicated for patients with hemophilia A with or without factor VIII inhibitors.
In May, Novo announced that Mim8 had achieved the co-primary endpoints in a Phase III trial, demonstrating that both once-weekly and once-monthly dosing of the treatment led to statistically significant reductions in treated bleeding episodes.
Outside of the US, Alhemo is currently approved in Australia, Japan, Switzerland and the European Union (EU), with specific indications varying by country, according to Novo.
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