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FDA grants orphan drug designation to APL-2 for autoimmune hemolytic anemia
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The FDA granted orphan drug designation to the C3 complement inhibitor APL-2 for the treatment of autoimmune hemolytic anemia.
Autoimmune hemolytic anemia refers to both cold agglutinin disease and warm autoimmune hemolytic anemia, which affect a combined 40,000 people in the United States and Europe.
No approved treatments exist for these rare autoimmune disorders, which are caused by pathogenic immunoglobulin G antibodies that react with red blood cells and can activate the complement system. This leads to premature destruction of red blood cells.
Patients with either disease can experience potentially life-threatening anemia or other acute complications.
APL-2 (Apellis Pharmaceuticals) is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds to C3 and C3b, blocking all three pathways of complement activation.
C3 is the protein at the center of the complement system. Targeting this protein may eliminate inappropriate or excessive C3 activation, protecting the cells against complement-mediated intravascular and extravascular destruction more effectively than other approaches.
“This orphan drug designation represents an important recognition by the FDA of APL-2’s potential to treat autoimmune hemolytic anemia,” Federico Grossi, executive vice president of clinical development and medical affairs for Apellis, said in a company-issued press release. “This is an important milestone as we build out our pipeline using the systemic administration of APL-2 in rare, complement-mediated diseases.”
A phase 2, open-label trial of APL-2 is underway to assess the efficacy, safety, tolerability and pharmacokinetics of multiple subcutaneous daily doses of the agent for patients with warm autoimmune hemolytic anemia or cold agglutinin disease.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.