FDA Approves Revolutionary (and Expensive) Hemophilia A Drug

On February 22, 2023, the FDA approved a new drug therapy, Altuviiio, for the treatment of Hemophilia A.

What is Hemophilia?

Hemophilia is an extremely rare genetic condition that prevents blood from clotting properly, leading to multiple serious health complications, including spontaneous bleeding.

Hemophilia is divided into type A and type B, with A occurring four times more often than B. Type A is affects approximately 1 in 5,000 male births each year.

Altuviiio is indicated for both adults and children and can be used for perioperative management, the treatment of routine prophylaxis, and control of spontaneous bleeding episodes.

It is the first hemophilia A treatment that delivers near to near-normal factor activity levels and significantly reduces bleeds in comparison to previous prophylactic therapies. The new drug, Altuviiio, is administered in a once-weekly dose with a half-life that’s three to four times greater than other Hemophilia A medications.

Expected Pricing of Altuviiio

Sanofi and Sobi manufacturers are co-developing Altuviiio, which is commercially available in April 2023.

Presently, there is little pharmacy pricing information available for Altuviiio. The manufacturers state that pricing will be “at parity” to the annual cost of prophylaxis treatment using Eloctate, which is now available at $12 per unit.

This works out to a weekly cost of $12,000 for a patient requiring 1,000 IUs, or an annual cost of $625,000. Some patients may require dosing as high as 3,000 IUs weekly, which would result in an annual cost of $1.8 million.

Analysts project that the drug’s sales will reach over $1.3 billion by 2029. Skyrocketing costs for prescription drugs is challenging many employers to discover hidden cost drivers on their prescription benefits.

For additional insights into the pharmaceutical market and drug pricing, check out this blog: PBMs Explained – What Are They and How Do They Relate to Soaring Pharma Costs?