Sweden-based Abliva has announced that its drug candidate NV354 has been received orphan drug designation (ODD) from the US FDA Office of Orphan Products Development to treat mitochondrial disease.

Sweden-based Abliva has announced that its drug candidate NV354 has been received orphan drug designation (ODD) from the US FDA Office of Orphan Products Development to treat mitochondrial disease.

Abliva is a clinical-stage company developing medicines to treat rare and severe primary mitochondrial diseases.

NV354 is an orally available compound being developed to treat severe primary mitochondrial diseases including Leigh syndrome.

Leigh syndrome is a rare disease mainly impacting the central nervous system.

Abliva CEO Ellen Donnelly said: “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.”

NV354 has indicated to be safe and efficacious in pre-clinical models, with regard to optimal pharmacological properties, including high levels of brain penetration.

This second programme in the Abliva portfolio, NV354 was developed by Abliva scientists.

NV354 complements the lead asset, KL1333, which also has received ODD.

Reference：

https://www.pharmaceutical-business-review.com/news/ablivas-nv354-receives-odd-us-fda/