Europe OKs Marketing of Hemophilia A Drug

The European Commission (EC) has granted marketing authorization for Altuvoct (efanesoctocog alfa, Sobi) to treat and prevent bleeds and for perioperative prophylaxis in hemophilia A for all age groups and disease severities. 

The EC also endorsed a recommendation by the European Medicines Agency (EMA) to support Altuvoct’s retention of orphan drug designation, granting a 10-year market exclusivity period. 

Hemophilia is a rare genetic condition in which the body underproduces or makes dysfunctional clotting protein factor VIII. It results in blood vessels being unable to heal as usual after injury.

The severity of symptoms ranges from mild to severe, depending on factor VIII levels and activity. Symptoms include easy bruising and inadequate clotting following mild injury. The disease affects around 0.7 in every 10,000 people in the EU.

The active substance in Altuvoct is efanesoctocog alfa, a recombinant human factor VIII, which works in lieu of factor VIII for successful clotting. 

The EC’s marketing authorization follows results from phase 3 studies evaluating the efficacy and safety of efanesoctocog alfa in adults and adolescents, and in children, who have severe hemophilia A.

The studies reported that once-weekly prophylaxis at 50 IU/kg provided significant bleed protection for all age groups, with 80%-88% of patients free from spontaneous bleeds.

They further found that the treatment provided mean factor VIII activity of more than 40 IU/dL for most of the week. Factor VIII activity levels of 40% and above are not considered hemophilic. Activity levels reached a low of 15 IU/dL by the end of the week and before the next dose; which is within the range of mild hemophilia.

Improvements in joint health, physical health, pain, and overall quality of life a year after starting treatment were reported.

Common side effects to efanesoctocog alfa include headache, vomiting, eczema, rash, urticaria, arthralgia, pain in extremity, back pain, and pyrexia. 

The drug should be used under the supervision of a physician experienced in treating hemophilia and will be available as 250 IU, 500 IU, 750 IU, 1000 IU, 2000 IU, 3000 IU, and 4000 IU powder and solvent for solution for injection. Use cases include on-demand and during surgical procedures.

Detailed recommendations for the use of Altuvoct will be described in the summary of product characteristics, which will be published in the European public assessment report in all official European Union languages. 

Annie Lennon is a medical journalist. Her writing appears on Medscape, Medical News Today, and Psych Central, among other outlets.