Roche announced Friday that ACE910 has been recognized as a breakthrough therapy by the U.S. Food and Drug Administration for treating those aged at least 12 years old diagnosed with hemophilia A with factor VIII inhibitors.
A rare genetic disorder, hemophilia A occurs when factor VIII, a protein essential for blood clotting, is defective or available in amounts insufficient in the body. Because of this, those with the disorder are at high risk of bleeding that's difficult to control or uncontrollable, including internal bleeding, particularly into the joints which prompts the need for replacements. The breakthrough therapy designation given to ACE910 is designed to speed up the development and review process for medicines showing early evidence in clinical trials that they offer substantial improvement in treatment compared to current options.
In a Phase 1 study, the hemophilia drug offered promise as a prophylactic treatment given as a weekly injection to patients with severe hemophilia A regardless of the presence of factor VIII inhibitors. Inhibitor development seriously complicates the condition, regardless of disease severity, making it hard, if not impossible, to meet sufficient levels of factor VIII enough to control bleeding using traditional replacement treatments. Since managing bleeding is still highly difficult, it showed that there was still a need for additional therapy options that patients can take advantage of.
"People with hemophilia A may require regular and frequent infusions of replacement clotting factor to reduce the risk of dangerous bleeding," said Sandra Horning, Global Product Development head and chief medical officer for Roche. Unfortunately, it is also possible for patients to develop inhibitors that can render replacements ineffective.
She added that Roche is pleased that ACE910 was given breakthrough therapy designation by the FDA, which recognizes that there is an unmet need involving patients with inhibitors. For more than 20 years, the company has been developing antibody treatments for blood disorders and is thrilled to expedite developing the hemophilia drug.
A Phase III trial for ACE910 is set to begin by the end of the year. It will involve patients with hemophilia A with factor VIII inhibitors while those without inhibitors will be part of a different Phase III trial scheduled for 2016. Another clinical trial will also commence in 2016 with pediatric patients as participants.
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