FDA finally approves Novo Nordisk’s once-daily hemophilia medication

Novo Nordisk got a once-daily hemophilia injection cleared days before 2025. Novo reported that the FDA approved its tissue factor pathway inhibitor (TFPI) antagonist concizumab, as a once-a-day regimen for treating or reducing the rate of bleeding episodes in people with hemophilia A or B with inhibitors aged 12 years and older. The prophylactic is contained in prefilled, premixed pens for subcutaneous administration; Novo said in the release that it would sell under the trade name Alhemo.
Currently, many treatments for hemophilia A or B with inhibitors use intravenous infusions, and Novo claims Alhemo is the only subcutaneous injection treatment for this specific patient group. Hemophilia with inhibitors is an acquired difficulty with the execution of the genetic bleeding disorder that hinders factor replacement treatments, a well-established category of treatments for the condition.
According to Novo Nordisk, between 30 percent and 5 to 10 percent of severe hemophilia A patients develop inhibitors that make treatment even more challenging. The company has projected about 800,000 people worldwide with this disease, while in the U.S. alone, there are about 32,000 patients.

Those in particular have been especially screwed – patients with hemophilia B with inhibitors – because until recently there was hardly anything in the way of prophylactic treatments for their particular type of condition that the company provided.
Alhemo, however, is out to neutralize a protein referred to as TFPI that helps prevent the formation of blood clots. In doing so, Alhemo is though to increase the reaction rate of thrombin, the protein that clots blood and prevents bleeding when the other clotting factors or absent or deficient when inhibited, Novo said.
The estimated mean ABR for patients under Novo’s drug was 1.7 compared to 11.8 among patients with no prophylaxis, and the median ABR of 0 for the treated spontaneous and traumatic bleeds compared with 9.8 ABR.

On a supportive secondary efficacy measure, 64% of patients who received Alhemo had no treated spontaneous and traumatic bleeds during the first 24 weeks after initiating treatment compared to 11% of those who did not receive prophylaxis over the same time duration.
Although Novo has successfully finally received approval from the legislator bodies for the sale of concizumab in the United States, the journey to this aim has not been smooth.
The FDA in January last year halted Novo’s trials over blood-clotting concerns that affected three patients. But Novo, which returned to its studies five months later, told Fierce Biotech in 2022 that the problems were history.
Novo executives disclosed on an earnings call that the therapy had received a complete response from the FDA on the dosing and manufacturing process of concizumab. Overall, till this point, the drug has had much less trouble outside the U.S. getting clear signals from places like Australia, Japan, Switzerland, and the EU to name but a few.
Still, as Novo did finally swoosh through the FDA’s desk with Alhemo before 2024, the Danish drug maker cannot keep up with Pfizer’s race to the hemophilia finish line.

Therefore, head-on competition between Novo and Pfizer’s products is rather improbable because the drugs are deployed for two different hemophilia patient groups: those with inhibitors and those without.
On the hemophilia front elsewhere, Novo is eager to challenge Roche’s primary bleeding disorder product Hemlibra which achieved nearly $4.8 billion in sales in the prior year. For the battle with Roche’s blockbuster Enjay, Novo is moving forward with Mim8, a bispecific antibody that links Factor IXa and X to replace missing Factor VIII as Hemlibra does.
Novo reported that its candidate met both the co-primary endpoints informing the phase 3 study – once-weekly Mim8 is associated with a statistically significant decrease in treated bleeding episodes as well as once-monthly Mim8.