CHMP Recommends Novo Nordisk’s Alhemo for Hemophilia A and B

Novo Nordisk's Alhemo (concizumab) has received a significant endorsement from the European Medicines Agency's human medicines committee for its use in preventing bleeding in haemophilia patients aged 12 and older. If it gains approval, Alhemo will become the first once-daily subcutaneous prophylactic treatment available for individuals with haemophilia A or B and inhibitors.

Haemophilia, affecting approximately 1,125,000 individuals worldwide, is a rare genetic disorder that hampers the body's ability to form blood clots, essential for stopping bleeding. Traditional treatments usually involve frequent intravenous infusions of clotting factor replacement products. However, an immune response to these clotting factors can lead to the development of inhibitors, complicating treatment. It is estimated that up to 30% of severe haemophilia A patients and up to 10% of severe haemophilia B patients develop these inhibitors.

Alhemo, developed by Novo Nordisk, is an anti-tissue factor pathway inhibitor monoclonal antibody. It is designed to aid in blood clot formation and prevent bleeding, even in the presence of inhibitors. The recent positive opinion from the Committee for Medicinal Products for Human Use (CHMP) was based on encouraging results from the late-stage explorer7 study. This study showed that Alhemo prophylaxis led to an 86% reduction in treated spontaneous and traumatic bleeds compared to no prophylaxis.

Novo Nordisk anticipates that the European Commission will make a final decision on Alhemo's approval within the next two months. Stephanie Seremetis, the chief medical officer for haemophilia at Novo Nordisk, hailed the positive opinion from CHMP as a "major milestone" for patients. She explained that Alhemo could significantly reduce the physical, emotional, and overall treatment burden for people with haemophilia. The medication is delivered in a pre-filled, multi-use, portable pen that can be stored at room temperature for up to four weeks, which could enhance patients' confidence in managing their daily activities. This is particularly crucial for those with haemophilia B and inhibitors, who currently have very limited treatment options.

Additionally, Novo Nordisk recently announced a multi-year partnership with NanoVation Therapeutics to advance genetic medicines for cardiometabolic and rare diseases. This collaboration, valued at $600 million, leverages Novo's expertise in cardiometabolic and rare diseases with NanoVation's proprietary long-circulating lipid nanoparticle technology for RNA delivery to cells outside the liver.

Overall, the potential approval of Alhemo represents a significant advancement in the treatment of haemophilia, offering a new once-daily subcutaneous option for patients who develop inhibitors. This development, combined with Novo Nordisk's recent partnership with NanoVation Therapeutics, underscores the company's commitment to innovation in addressing complex medical challenges.