EMA Approves Factor VIII Deficiency Clotting Drug

The European Medicines Agency (EMA) this week recommended granting a marketing authorization for Altuvoct (efanesoctocog alfa) for the treatment and prophylaxis of bleeding in patients with hemophilia A caused by factor VIII deficiency.

Patients with this genetic disorder lack a specific clotting protein in the blood that means injured blood vessels cannot heal in the normal way. The severity of symptoms ranges from mild to severe depending on the amount of factor VIII present in the blood and its activity.

The active substance of Altuvoct is efanesoctocog alfa, a recombinant human factor VIII, which replaces the missing coagulation factor VIII needed for effective hemostasis.

Suitable for Adults and Children

At its April 25 meeting, the EMA's Committee for Medicinal Products for Human Use (CHMP) accepted evidence from clinical trials that Altuvoct could prevent and control bleeding when used on demand and during surgical procedures in adults and children with hemophilia A.

The most common side effects with Altuvoct are headache, vomiting, eczema, rash, urticaria, arthralgia, pain in extremity, back pain, and pyrexia (fever).

Altuvoct 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.

The full indication for Altuvoct is treatment and prophylaxis of bleeding in patients with hemophilia A (congenital factor VIII deficiency) of all age groups.

Treatment with Altuvoct should be under the supervision of a physician experienced in the treatment of hemophilia, the CHMP stressed.

The EMA designated Altuvoct as an orphan medicine during its development. It is now up to the European Commission to decide whether to grant a marketing authorization for Altuvoct to the applicant, Swedish Orphan Biovitrum AB.