MHRA Approves Sobi's Altuvoct for Severe/Moderate Haemophilia A

The Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval for Sobi and Sanofi's drug, efanesoctocog alfa, to treat haemophilia A in patients two years of age and older. This approval, under the brand name Altuvoct, authorises its use to treat and prevent bleeding episodes in individuals with moderate to severe forms of this genetic bleeding disorder.

Haemophilia A is a rare condition caused by a deficiency in factor VIII, a crucial protein for blood clotting. This deficiency can lead to frequent bleeding episodes, posing risks such as pain, joint damage, and potentially life-threatening haemorrhages. Altuvoct is an intravenous medication designed to replace the missing factor VIII, thereby aiding blood clotting and offering temporary control over bleeding tendencies.

The MHRA's decision to approve Altuvoct was heavily influenced by data from the pivotal XTEND-1 study, which involved 159 participants aged 12 and above with severe haemophilia A. Over the course of this year-long study, weekly administrations of Altuvoct resulted in 65% of participants experiencing no bleeding episodes at all, while the remaining 35% reported a marked decrease in such episodes. These findings demonstrate the drug's significant potential in managing this serious condition.

Further supporting this approval were results from the phase 3 XTEND-Kids trial, which studied 74 children under the age of 12 with severe haemophilia A. Additionally, data from a modelling study addressing moderate cases of the disorder reinforced the drug's efficacy and safety profile.

Julian Beach, interim executive director of healthcare quality and access at the MHRA, expressed confidence in the approval, stating: "Patient safety is our top priority, which is why I am pleased to confirm approval of [Altuvoct] to treat and prevent bleeding in patients two years and above with severe or moderate haemophilia A. We’re assured that the appropriate regulatory standards of safety, quality, and efficacy for the approval of this new formulation have been met." He also emphasized that the product's safety will continue to be closely monitored.

The collaboration between Sanofi and Sobi involves joint efforts in the development and marketing of efanesoctocog alfa. In the United States, the drug is marketed under the name Altuviiio. While Sobi retains the rights for final development and commercialisation across Europe, North Africa, Russia, and most Middle Eastern markets, Sanofi holds these rights in North America and all other global regions, excluding those under Sobi's purview.

In summary, the MHRA’s approval of Altuvoct represents a promising advancement for individuals living with haemophilia A, offering a new therapeutic option that has demonstrated significant efficacy in reducing bleeding episodes while maintaining a strong safety profile.