Roche Haemophilia Drug Gets Breakthrough FDA Designation

Roche Haemophilia Drug Gets Breakthrough FDA Designation
Roche Office Rastatter / Wikimedia Commons

Roche is very proud to announce that the U.S. Food and Drug Administration (FDA) has recently granted a breakthrough therapy designation to its investigational medicine, ACE910 (RG6013, RO5534262). This is meant to be a prophylactic treatment to people ages 12 years or older who are suffering from haemophilia A with factor VIII inhibitors.

Haemophilia A is said to be a “rare genetic disorder” wherein a person does not possess enough factor VIII in the body. Factor VIII is actually a blood clotting protein and when a person suffers deficiency from this, he or she may have difficulty to control one’s bleeding. This may also happen internally and through the joints, which may lead the person to require joint replacements. A Phase 1 study of the ACE910 showed that the said prophylactic treatment may have promising results in treating patients with haemophilia A when administered weekly via subcutaneous injection.


The breakthrough therapy designation given by the FDA means that the both the development and review of the ACE910 can be accelerated. Moreover, this designation is granted to medicines that are able to show “substantial improvement” over present treatment options for serious diseases through early clinical findings.

Roche Chief medical Officer and Head of Global Product Development Sandra Horning, M.D. admits that Roche has actually been working on antibody treatments for people suffering from blood disorders for more than 20 years. And the company, certainly, could not happier with the new breakthrough designation of the ACE910.

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Currently, Roche is preparing to hold a Phase III trial of ACE910 among patients with haemophilia A with factor VIII inhibitors sometime during the end of 2015. Meanwhile, another Phase III trial is also being scheduled for 2016. This one will be for patients without inhibitors. At the same time, a trial involving pediatric patients with haemophilia A is also expected to be conducted in 2016.

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