Home / FDA News / Apellis’ APL-2 Receives Fast Track Designation from the FDA for the Treatment of Paroxysmal Nocturnal Hemoglobinuria

Apellis’ APL-2 Receives Fast Track Designation from the FDA for the Treatment of Paroxysmal Nocturnal Hemoglobinuria

WALTHAM Mass., and CRESTWOOD, Ky., Feb. 11, 2019 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals 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. 

About Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, potentially life-threatening disease characterized by complement-mediated hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes and/or some degree of bone marrow dysfunction. A significant subset of patients treated with the current standard of care still suffer from debilitating anemia and transfusion dependence.

About APL-2 
APL-2 is designed to inhibit the complement cascade centrally at C3 and may have the potential to treat a wide range of complement-mediated diseases more effectively than is possible with partial inhibitors of complement. APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer that binds specifically to C3 and C3b, effectively blocking all three pathways of complement activation (classical, lectin, and alternative). To date, APL-2 has generally been well-tolerated. No significant infections have been observed in trials involving the systemic administration of APL-2, including the trials in PNH, AIHA or other trials.

Clinical trials 
In hematologic diseases, Apellis is currently evaluating APL-2 in PEGASUS, a Phase 3 trial for patients with PNH as well as in two Phase 1b trials (PHAROAH and PADDOCK) for systemic administration. Previously reported interim data from these 1b trials showed improvements in lactate dehydrogenase and hemoglobin levels in patients who are suboptimal responders to eculizumab and untreated patients, respectively. Apellis is also testing APL-2 for systemic administration in a Phase 2 clinical trial in autoimmune hemolytic anemia (AIHA) and a Phase 2 clinical trial in complement dependent nephropathies. For additional information regarding our clinical trials, visit www.apellis.com/clinical-trials.html.

About Apellis
Apellis Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds for the treatment of a broad range of life-threatening or debilitating autoimmune diseases based upon complement immunotherapy through the inhibition of the complement system at the level of C3. Apellis is the first company to advance chronic therapy with a C3 inhibitor into clinical trials. For additional information about Apellis and APL-2, please visit http://www.apellis.com.

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