World News: 12:00 GMT Monday 11th February 2019. [Apellis Pharmaceuticals, Inc. via Globe Newswire via SPi World News]
WALTHAM Mass., and CRESTWOOD, Ky., Feb. 11, 2019 (GLOBE NEWSWIRE) -- Inc., (Nasdaq:APLS) a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat disease through the inhibition of the complement system, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the Company’s APL-2, a novel inhibitor of complement factor C3, for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). APL-2 is currently being studied in a Phase 3 active comparator trial in patients with PNH who remain anemic despite treatment with eculizumab.
This new Fast Track designation for APL-2 for all patients with PNH, supersedes the prior Fast Track designation granted on December 15, 2016 for APL-2 for the subset of PNH patients who continue to experience hemolysis requiring blood transfusions despite receiving therapy with eculizumab.
The FDA’s Fast Track program facilitates the development and expedites the review of drugs to treat serious conditions and fill an unmet medical need, allowing important new drugs to reach the patient earlier. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may have potential benefits over existing therapies. Drugs that receive a Fast Track designation are eligible for more frequent meetings and written communication with the FDA to discuss development plans and clinical trial design. In addition, if criteria are met, Fast Track-designated drugs are eligible for Rolling Review, where a drug company can separately submit sections of its New Drug Application to the FDA, and Accelerated Approval and Priority Review, in which drugs for serious conditions fulfilling an unmet medical need can be approved based on a surrogate endpoint.
“We are very pleased that the FDA recognizes the potential for APL-2 to help every patient with PNH. We believe that APL-2 has the potential to be a best in class treatment for all PNH patients,” said Pascal Deschatelets, PhD, co-founder and chief operating officer of Apellis.
Globe Newswire: 12:00 GMT Monday 11th February 2019
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