Neurophth's gene therapy granted orphan drug designation

By The Science Advisory Board staff writers

September 25, 2020 -- Neurophth Therapeutics' lead candidate, NR082, an adeno-associated virus (AAV)-based gene therapy, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of Leber hereditary optic neuropathy associated with an ND4 mutation.

NR082 is an ophthalmic AAV-based gene therapy. It uses an AAV2 vector to express the human ND4 gene in the retinal ganglion cells to repair optic neuropathy caused by the mutation. Investigator-initiated trials began in 2011, which demonstrated the long-term safety, effectiveness, and durability of AAV gene therapy in clinical settings.

The disease is a maternally inherited mitochondrial disease that causes vision loss. It impacts 1-9:100,000 people worldwide but has no effective treatments. Around 70%-90% of cases are caused by the ND4 mutation harboring a point mutation at nucleotide 11778 associated with a G-to-A transition.

Orphan drug designation provides opportunities for grant funding, fast approval channels, and some incentives such as waiver of new drug application fees, tax credits for clinical trial expenses, and exemption for prescription drug users' fees.

Neurophth is actively preparing the China/U.S. investigational new drug (IND) applications and plans to carry out the registration clinical Phase I/II/III registration trial to evaluate the safety, efficacy, and durability of NR082.


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