Abliva AB (Nasdaq Stockholm: ABLI), a clinical-stage company developing medicines for the treatment of rare and severe primary mitochondrial diseases, today announced that the drug candidate NV354 has been granted Orphan Drug Designation (ODD) for the treatment of mitochondrial disease by the United States FDA Office of Orphan Products Development.
Abliva’s orally available compound NV354 is being developed for the treatment of severe primary mitochondrial diseases including Leigh syndrome, a rare disease primarily affecting the central nervous system.
“The granting of ODD to NV354 by the U.S. FDA is a validation of the quality of the NV354 pre-clinical program and another important milestone for Abliva. The ODD will be beneficial to us as we work to progress NV354 and evaluate the compound in patients who are in urgent need of therapies to relieve their suffering,” said Ellen Donnelly, CEO at Abliva.
In pre-clinical models, NV354 has been shown to be safe and efficacious with optimal pharmacological properties, including high levels of brain penetration. NV354, the second program in the Abliva portfolio, was developed by Abliva scientists and complements the lead asset, KL1333, which also has ODD.
Orphan drug designation will facilitate regulatory and scientific advice meetings with the FDA regarding the NV354 program and may enable a focused development program with an expedited approval process. If approved, ODD also provides market exclusivity for NV354 (seven years in the U.S.) once the drug is approved, which adds to the protection provided by the NV354 patent estate.