The Swiss drugmaker had submitted a supplemental biologics license application to the US regulator, covering the data recorded from the phase 3 Haven 3 study.
Hemlibra’s priority review follows the breakthrough therapy designation granted by the FDA in mid April. The regulator is expected to take a call on the drug approval by 4 October 2018.
A bispecific factor IXa- and factor X-directed antibody, Hemlibra has been designed to be a prophylactic (preventative) treatment. It is intended to be administered weekly once by an injection of a ready-to-use solution subcutaneously.
Roche chief medical officer and global product development head Sandra Horning said: “People with haemophilia A can face significant challenges in managing their condition and may need to adapt their daily lives to avoid bleeds and accommodate treatment.
“We believe the FDA’s decision to grant Priority Review to Hemlibra underscores its potential to improve the standard of care for people without factor VIII inhibitors and to help reduce treatment burden by offering more flexible subcutaneous dosing options.”
The Haven 3 study was held in 152 patients, aged 12 years or more, with haemophilia A without inhibitors, who were treated in the past with factor VIII therapy on-demand or for prophylaxis in the trial.
The randomized, multicentre, open-label study assessed the efficacy, safety and pharmacokinetics of Hemlibra prophylaxis against no prophylaxis (episodic/on-demand factor VIII treatment).
Hemlibra prophylaxis, which was subcutaneously dosed every week or every two weeks, showed a statistically significant and clinically meaningful decrease in treated bleeds, in comparison to no prophylaxis in Haven 3.
The drug was approved by the FDA in last November for routine prophylaxis to prevent or decrease the frequency of bleeding episodes in adults and children with haemophilia A with factor VIII inhibitors.
In February this year, Hemlibra was approved by the European Commission for routine prophylaxis of bleeding episodes in patients with haemophilia A with factor VIII inhibitors.
Image: Hemlibra was developed by Genentech, a member of the Roche Group. Photo: courtesy of Business Wire.