Abliva Receives Orphan Designation from the European Commission for its Drug Candidate NV354

Abliva AB (Nasdaq Stockholm: ABLI) a clinical-stage company developing medicines for the treatment of rare and severe primary mitochondrial disease, today announced that the European Commission has granted orphan designation for the company’s drug candidate NV354 for the treatment of Leigh syndrome, facilitating its clinical development in neurological mitochondrial disease.

“The orphan designation by both the European Commission and the US FDA is a great recognition of the NV354 program and the first-in-class prodrug technology for the treatment of pediatric Leigh syndrome. This will facilitate the future progression to clinical development of a much-needed treatment for these patients”, said Ellen Donnelly, CEO at Abliva.

Abliva’s drug candidate NV354 is being developed for mitochondrial disease with neurologic complications, including Leigh syndrome, MELAS, and LHON. The program’s preclinical development has been completed, and in April 2023, the company was granted orphan drug designation (ODD) for NV354 in the US for the treatment of mitochondrial disease.

Abliva has now received orphan designation for NV354 also from the European Commission for the treatment of Leigh syndrome. Orphan designation in Europe offers Abliva scientific advice on study protocols, various fee reductions, and access to EU grants. If approved for EU and US orphan (drug) status when authorized for marketing, NV354 would benefit from ten years of market exclusivity within the EU and seven years within the US.