FDA expands Alhemo approval to help more people with hemophilia
Preventive therapy now indicated for those who don't have inhibitors
The U.S. Food and Drug Administration (FDA) has expanded its approval of Alhemo (concizumab-mtci), a daily therapy injected under the skin for preventing or reducing the frequency of bleeding episodes in people with hemophilia A or B, ages 12 and older.
Sold by Novo Nordisk, Alhemo had been approved in the U.S. only for patients with inhibitors, that is, neutralizing antibodies that can lower the effectiveness of standard hemophilia treatments. Now, the preventive therapy is also indicated for those who don’t have inhibitors.
“The FDA approval of an expanded indication for Alhemo marks a meaningful step forward for people with hemophilia A or B without inhibitors who are looking for a new prophylaxis treatment option,” Anna Windle, PhD, senior vice president, clinical development, medical and regulatory affairs at Novo Nordisk, said in a company press release. “By building on the initial indication for Alhemo for those with hemophilia with inhibitors — an especially significant development in hemophilia B where challenges still exist despite advanced treatment options — Novo Nordisk continues its 35+ year legacy in rare bleeding disorders to continue to help address the unmet needs of this community.”
Hemophilia is a bleeding disorder where there’s a lack or dysfunction of certain clotting proteins, specifically factor VIII (FVIII) in hemophilia A and factor IX (FIX) in hemophilia B.
Factor replacement therapies are standard treatments where a version of the missing or dysfunctional protein is administered to restore blood clotting. Some patients develop antibodies against the clotting factors, however, which can render them less effective.
What is Alhemo and how does it work in hemophilia?
Available in prefilled, pre-mixed pens, Alhemo blocks the activity of a protein called tissue factor pathway inhibitor (TFPI) that normally acts as a quality-control mechanism to help prevent clotting. By inhibiting it, Alhemo can help promote clotting and, because this mode of action doesn’t rely on FVIII or FIX, Alhemo can be used in patients who’ve developed inhibitors against those clotting factors.
Alhemo’s approval for patients without inhibitors was based on data from explorer8 (NCT04082429), a Phase 3 clinical trial that enrolled nearly 150 people with hemophilia A or B, ages 12 and older, who didn’t have inhibitors. The participants were either treated with Alhemo or not given a preventive therapy.
The annualized bleeding rate for spontaneous and traumatic bleeding episodes was significantly lower with Alhemo than with no prophylaxis, by a mean of 86% for people with hemophilia A and by 79% for those with hemophilia B. Notable reductions in mean and median annualized bleeding rates were also seen with Alhemo in patients with hemophilia A and B. The most common side effects were injection site reactions and headache.
“For people living with hemophilia, it is important to continually monitor and discuss bleed control with their healthcare professional,” said Allison P. Wheeler, MD, scientific director of the Washington Center for Bleeding Disorders in Seattle. “With today’s approval of Alhemo for hemophilia A or B without inhibitors, more people living with these rare blood disorders now have a daily prophylaxis option that may help decrease their bleeding rates.”
Alhemo is approved in the European Union for people with hemophilia A or B, ages 12 and older, with inhibitors. A request for its approval for patients without inhibitors is currently under review, with a decision expected in the coming months.
About the Author
