Sickle Cell Therapy Candidate Recommended for European Orphan Drug Designation

La Jolla’s investigational therapy LJPC-401 (synthetic human hepcidin) to treat sickle cell disease has been recommended for orphan drug designation through a positive opinion issued this week by the European Medicines Agency‘s (EMA) Committee for Orphan Medicinal Products (COMP).

“We are encouraged by the positive feedback and continued support of the European regulatory authorities,” George F. Tidmarsh, MD, PhD, president and CEO of La Jolla, said in a press release.

“Following our recently reported positive results from our Phase 1 study of LJPC-401, which demonstrated a clear, dose-dependent effect of LJPC-401 on serum iron, and our reaching of an agreement with the EMA on study design, we look forward to initiating our pivotal study of LJPC-401 in mid-2017,” Tidmarsh said.

Hepcidin is a protein that regulates the entry of iron into blood circulation. An endogenous peptide hormone, hepcidin halts the over-accumulation of iron in vital organs, including the liver and heart, where too much iron can cause damage and even death.

La Jolla is developing LJPC-401 to potentially treat iron overload, which occurs in sickle cell disease (SCD). Last year, the COMP designated LJPC-401 as an orphan medicinal product to treat beta thalassemia intermedia and major (a blood disorder that reduces the production of hemoglobin).

SCD is a common hereditary form of anemia in which a mutated form of hemoglobin distorts the red blood cells into a sickle (crescent) shape and impairs blood delivery. Severe SCD can cause life-threatening chronic hemolytic anemia, stroke, and serious problems in the lungs, spleen, kidney, and other organs.

With chronic hemolytic anemia, hepcidin levels may also be suppressed, which could lead to iron overload. Currently, the only treatment approved for iron overload are iron chelators; however, they may lead to kidney failure, liver failure, or gastrointestinal hemorrhage.

While LJPC-401 is being developed to treat iron overload, it may also  reduce red cell hemoglobin concentration leading to less sickle cell formation.

If LJPC-401 is eventually granted orphan drug status, La Jolla will likely benefit from incentives provided by the EU to develop the medicine faster and less expensively than typical drug development. The designation is granted to develop drugs that address diseases with significant unmet needs, those that are of major public concern, or those deemed too rare to justify major financial investment by drug developers.