September 25, 2020
1 min read

FDA grants orphan drug designation for LHON gene therapy

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

We were unable to process your request. Please try again later. If you continue to have this issue please contact

Neurophth Therapeutics has received orphan drug designation from the FDA for NR082 for the treatment of Leber hereditary optic neuropathy associated with ND4 mutation, according to a press release.

Between 70% and 90% of LHON cases are caused by ND4 mutation, according to the release.

“[NR082] uses recombinant adeno-associated virus serotype 2 to deliver the genetically modified ND4 gene. After a single intravitreal injection, the gene is translated and expressed in cells, which effectively supplements the function loss caused by endogenous mutation,” Alvin Luk, PhD, MBA, CCRA, CEO of Neurophth, said in the release. “Through this gene therapy, the electron transport function of mitochondrial respiratory chain was maintained, and the increase of ATP synthesis restored the normal function of mitochondria, which in turn improved the sensory function of the retinal ganglion cells and improved the visual acuity of LHON patients.”

The company is preparing investigational new drug applications in China and the U.S. and is planning to begin clinical trials to evaluate safety, efficacy and durability of NR082.