FDA Grants Orphan Drug Status to AMP-L2.7.D7 for Treatment of Congenital Erythropoietic Porphyria

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AMP-L2.7.D7 (ciclopirox) for treatment of Congenital Erythropoietic Porphyria (also known as Gunther’s disease).

AMP-L2.7.D7, developed by Atlas Molecular Pharma, is a pharmacological chaperone — a molecule that binds to uroporphyrinogen III synthase, the defective enzyme in Congenital Erythropoietic Porphyria. This is one of the enzymes in the heme biosynthetic pathway. Heme, an essential component to life, is part of hemoglobin, the protein that transports oxygen in red blood cells.

Deficiency of this enzyme leads to the accumulation of porphyrins in several tissues in the body, triggering the disease symptoms. Binding of AMP-L2.7.D7 to the defective enzyme improves its stability and helps reverse the disease symptoms.

The European Medicines Agency and the European Commission previously approved ciclopirox as an Orphan Medicinal Product to treat Congenital Erythropoietic Porphyria.

“The favourable report from the European Medicines Agency and the approval of ciclopirox as an orphan drug by the European Commission and the FDA are very important steps in the development of ciclopirox for the treatment of Congenital Erythropoietic Porphyria,” Emilio Díez, CEO and CSO of Atlas Molecular Pharma, said in a press release.

“ATLAS is currently securing appropriate funding for the clinical trials that demonstrate the benefit of ciclopirox in the treatment of this devastating disease. The new OMP [Orphan Medicinal Product] and ODD [Orphan-Drug designation] status will facilitate the process and ultimately contribute to our goal of delivering ciclopirox to patients suffering from Congenital Erythropoietic Porphyria at the earliest time possible,” Diez said.

ATLAS Molecular Pharm is a private company founded in 2015 in Derio, Spain, as a spin-off from the Centre for Co-operative Research in Biosciences (CIC bioGUNE).

“Understanding the molecular mechanism of this disease has enabled us to design a therapy based on pharmacological chaperones, molecules that bind to the defective protein fixing its stability problem and reversing its pathogenic effects,” said Oscar Millet, Director of the Laboratory of Protein Stability and Inherited Disease of the CIC bioGUNE.

The orphan drug designations granted by the FDA and European Medicines Agency are for drugs and biologics being developed as therapies for rare diseases. The designation opens several incentives, including tax credits and exemption of several fees.

Additionally, Atlas will receive scientific advice and high-quality, clinical trial protocol assistance that will help the company launch clinical trials for congenital erythropoietic porphyria patients in the future.