Electra Therapeutics Receives FDA Orphan Drug Designation for ELA026 for the Treatment of Hemophagocytic Lymphohistiocytosis (HLH)

ELA026 has the potential to address a high unmet need for patients with a rare, life-threatening hyperinflammatory disease

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Electra Therapeutics, Inc., a clinical stage biotechnology company developing antibody therapies against novel targets for immunological diseases and cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ELA026 for the treatment of hemophagocytic lymphohistiocytosis (HLH). ELA026, a first-in-class antibody therapy targeting signal regulatory proteins (SIRP), is a novel approach to treat secondary HLH (sHLH), a rare, life-threatening disease for which there is no approved treatment. sHLH can be triggered by cancer, infection, autoimmune disease, or immunotherapy, and is associated with a severe inflammatory response (a cytokine storm) for which patients require immediate intervention.

“The Orphan Drug Designation is an important milestone in ELA026’s clinical development in sHLH,” said Kim‑Hien Dao, DO, PhD, Chief Medical Officer of Electra Therapeutics. “This designation underscores the high unmet medical need in sHLH and supports our continued efforts to evaluate the effectiveness of ELA026 in depleting the pathogenic immune cells responsible for the cytokine storm and addressing the high early mortality observed in this disease.”

The FDA grants ODD status to investigational therapies intended to treat diseases that affect fewer than 200,000 people in the United States. This designation enables certain incentives, including tax credits for qualified clinical trials, prescription drug user-fee exemptions, and potential seven-year marketing exclusivity upon FDA approval.

About Secondary Hemophagocytic Lymphohistiocytosis

Secondary hemophagocytic lymphohistiocytosis (sHLH) is a rare, life-threatening hyperinflammatory disease for which there is no approved treatment. It can be triggered by cancer, infection, autoimmune disease, or immunotherapy. sHLH is associated with a severe inflammatory response for which patients require immediate intervention. Without effective treatment, patients may experience multiple organ failure and death. sHLH has a high mortality rate during the first months of diagnosis, with malignancy-associated HLH (mHLH) patients having the poorest outcomes.

About Electra Therapeutics

Electra Therapeutics is a clinical stage biotechnology company developing therapies against novel targets for immunological diseases and cancer. The company’s lead product candidate, ELA026, is a first-in-class monoclonal antibody that targets SIRP on the cell surface of myeloid cells and T lymphocytes, and selectively depletes pathological immune cells. ELA026 is in clinical development for secondary hemophagocytic lymphohistiocytosis (sHLH), a rare, life-threatening hyperinflammatory condition for which there is no approved treatment, as well as additional disease indications. For more information, please visit www.electra-therapeutics.com and follow us on LinkedIn.