Concizumab-mtci

Novo Nordisk’s Alhemo (concizumab-mtci) has been approved by the US Food and Drug Administration (FDA) to prevent or reduce bleeding episodes in a broader population of haemophilia A and B patients. The therapy has now been authorised for use as a once-daily prophylaxis in adult and paediatric patients aged 12 years and older with haemophilia A or B without inhibitors, building on its existing indication for haemophilia A or B patients with inhibitors. Affecting approximately 800,000 people globally, haemophilia is a rare inherited bleeding disorder that impairs the body’s ability to make blood clots. The condition is usually treated with regular intravenous infusions of clotting factor replacement products. However, some patients can develop inhibitors as an immune response to the clotting factors in these therapies, limiting treatment options. Administered as a subcutaneous injection, Novo’s Alhemo is a tissue factor pathway inhibitor antagonist that is designed to help the blood to clot and prevent bleeding, regardless of inhibitor status. Anna Windle, senior vice president, clinical development, medical and regulatory affairs, Novo Nordisk, said: “The FDA approval of an expanded indication for Alhemo marks a meaningful step forward for people with haemophilia A or B without inhibitors who are looking for a new prophylaxis treatment option.” The regulator’s latest decision on the therapy was based on results from the late-stage explorer8 trial, which demonstrated a statistically significant reduction in annualised bleeding rate (ABR) of 79% for haemophilia B patients without inhibitors and 86% for haemophilia A patients without inhibitors, compared to no prophylaxis. There were also notable reductions in the average and median ABRs, which Novo said “further reinforced the efficacy of Alhemo prophylaxis in reducing bleeds across both patient groups”. Also welcoming the decision, Allison Wheeler, scientific director, Washington Center for Bleeding Disorders, said: “For people living with haemophilia, it is important to continually monitor and discuss bleed control with their healthcare professional. “With [this] approval of Alhemo for haemophilia 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.”

The FDA cleared additional indications for Novartis’s cholesterol-lowering drug Leqvio (inclisiran) and Novo Nordisk's haemophilia prophylactic Alhemo (concizumab-mtci), broadening their use to wider patient populations.Leqvio gets go-ahead as montherapyNovartis's twice-yearly injectable siRNA drug Leqvio was approved by the FDA as monotherapy alongside diet and exercise to lower low-denisty lipoprotein cholesterol (LDL-C) in adults with hypercholesterolaemia, thereby eliminating the previous requirement for concurrent statin therapy.“With this new indication enabling Leqvio's use as monotherapy…we now have the potential to help even more patients achieve their LDL-C lowering goals earlier in their treatment journey," said Victor Bultó, president of Novartis US.The US regulator said Leqvio’s broader indication was approved based on strong LDL-C-lowering data from PCSK9-targeting therapies. Additional label updates include replacing "primary hyperlipidaemia" terminology with the more specific "hypercholesterolaemia" designation throughout the prescribing information.Novartis noted the decision also aligns with the 2025 American College of Cardiology/American Heart Association Clinical Practice Guidelines, recommending more aggressive treatment approaches for patients with acute coronary syndrome.Leqvio was discovered by Alnylam and co-developed with The Medicines Company, which Novartis acquired in 2020 for $9.7 billion. First approved in the EU for hypercholesterolaemia or mixed dyslipidaemia in 2020, the siRNA’s US approval faced initial delays owing to manufacturing issues, but was subsequently cleared by the FDA in 2021 for patients on maximally tolerated statin therapy.The Swiss drugmaker expects Leqvio not only to surpass $1 billion in sales this year, but potentially outperform consensus forecasts long term. For more, read – Spotlight On: Novartis' fastest growing drugs.Alhemo cleared for patients without inhibitorsThe FDA also okayed an expanded indication for Novo Nordisk's Alhemo (concizumab-mtci), extending its prophylactic use to adult and paediatric patients aged 12 and older with haemophilia A or B without inhibitors. The latest approval builds upon the tissue factor pathway (TFPI) inhibitor antagonist’s approval last year for patients with inhibitors after being rejected by the agency on the first attempt.Anna Windle, senior VP of clinical development, medical & regulatory affairs at Novo Nordisk, said the latest approval “marks a meaningful step forward for people with haemophilia A or B without inhibitors who are looking for a new prophylaxis treatment option."The label expansion was supported by the Phase III explorer8 study, wherein Alhemo achieved an 86% reduction in annualised bleeding rate for patients with haemophilia A and 79% reduction for those with haemophilia B versus no prophylaxis.Following a positive recommendation by the EU drug advisory body last week, the anti-TFPI monoclonal antibody is also on the verge of approval in Europe for the same indication.With the additional indication, Alhemo emerges as a potential rival to Pfizer’s once-weekly Hympavzi (marstacimab-hncq), approved last year for patients without inhibitors. Pfizer also seeks Hympavzi’s expansion to patients with inhibitors following a successful Phase III study.