Will Sanofi's Altuviiio transform Hemophilia treatment?

Sanofi's once-weekly hemophilia A treatment Altuviiio (efanesoctocog alfa) has been designated an orphan drug in Korea, accelerating its introduction.

Medical and industry insiders are paying keen attention to see if it will change hemophilia treatment, as clinical trials have shown an extended dosing cycle.

The Ministry of Food and Drug Safety (MFDS) designated Altuviiio as an orphan drug on May 3. Altuviiio is a gene recombinant hemophilia A treatment developed by Sanofi that extends the dosing cycle by increasing the half-life. It is the first time in six years that a congenital hemophilia A treatment has been designated as an orphan drug in Korea since Hemlibra (emicizumab).

Altuviiio was approved by the U.S. Food and Drug Administration (FDA) in February last year as an “on-demand treatment” and “routine prophylaxis” for the control and prevention of bleeding and perioperative management of adult and pediatric patients with hemophilia A. It is licensed and marketed in Japan, Taiwan, and other countries.

It has not yet been approved for marketing authorization in Korea and is currently undergoing a bridging study in Korea, including Korean patients. The orphan drug designation allows Sanofi to obtain conditional authorization based on phase 2 data and accelerate the launch of Alltuviiio in Korea.

The orphan drug designation is based on a phase 3 clinical trial (XTEND-1). Results showed a significant 77 percent reduction in annualized bleeding rates (ABR) in the Altuviiio arm (p<0.001) compared to prophylaxis with an existing factor VIII agent.

In addition, the mean weekly factor activity in the Altuviiio arm was greater than 40 IU/dL, with a mean of 15 IU/dL at Day Seven. Altuviiio was well tolerated, with no antibody events in the Altuviiio arm. The most common adverse reactions associated with Altuviiio treatment were headache, arthralgia, falls, and back pain.

The medical experts who previously reviewed the orphan drug designation also expressed positive opinions about Altuviiio's efficacy and safety. The Central Pharmacy Review Committee met remotely on April 9. It concluded that the number of patients, safety, and efficacy based on the submission and other factors warranted orphan drug designation for Altuviiio.

The review noted that Altuviiio has a standard half-life that is three to four times longer than other medicines, allowing for longer dosing intervals, and that it has been shown to maintain factor VIII activity at least 10 percent higher for seven days. Panelists reviewing the submission also saw evidence for Altuviiio’s efficacy in preventing and treating bleeding.

"From the patient's point of view, the reduction in dosing is very important," a committee member said. "Side effects were reduced, costs were lowered, and there were no significant adverse event reports."