Neurophth Therapeutics, Inc., today announced that its leading candidate, NR082 (rAAV2-ND4, NFS-01 project), was granted an orphan drug designation (ODD) by the U.S. FDA for the treatment of Leber's Hereditary Optic Neuropathy associated with ND4 mutation.
Leber's Hereditary Optic Neuropathy (LHON) is a maternally inherited mitochondrial disease, characterized by acute or subacute, painless vision loss or even loss simultaneously or sequentially, accompanied by central visual field defect and color vision impairment with poor prognosis. It was first reported by German scholar Leber in 1871. It affects about 1-9:100,000 people worldwide. LHON is one of the blinding diseases. The disease mainly occurs in young- and middle-aged men. Currently, there is no effective treatment for LHON. About 70% - 90% of LHON is caused by ND4 mutation of harboring a point mutation at nucleotide 11778 associated with a G-to-A transition. With the development of NR082, AAV-based gene therapy of LHON becomes possible.
"Due to the lack of effective treatment, the quality of life of LHON patients associated with ND4 mutation is very poor, and a huge unmet medical needs have not been fulfilled," said Dr. Alvin Luk, Chief Executive Officer at Neurophth. "NR082 is the first candidate drug developed by Neurophth. It uses recombinant adeno-associated virus serotype 2 to deliver the genetically modified ND4 gene (rAAV2-ND4). After a single intravitreal injection, the gene is translated and expressed in cells, which effectively supplements the function loss caused by endogenous mutation. 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."
Luk added, "the significance of orphan drug designation is that regulators recognize the unmet medical needs of rare diseases like LHON. The recognition of NR082 will reduce the R&D investment to a certain extent and accelerate the progress of clinical trials and marketing registration. Furthermore, Neurophth is committed to fundamentally solve the causes and change the quality of life of patients through a single treatment of gene therapy."
Professor Bin Li, Founder, Chairman and Chief Scientific Officer at Neurophth, said: "NR082 has been granted as orphan drug by U.S. FDA, which further strengthens our focus on gene therapy for rare ophthalmic diseases, and develops more drugs for treatment of ocular genetic diseases, bringing hope to patients with ocular genetic diseases in the world".
*FDA grants orphan drug designation to drugs and biological products designed to safely and effectively treat, diagnose, or prevent rare diseases or conditions affecting less than 200,000 people in the US. According to the Orphan Drug Act of FDA, Orphan Drug Designation (ODD) provides opportunities for grant funding, fast approval channel, and some incentives, such as waiver of New Drug Application (NDA) fees, tax credits for clinical trial expenses and exemption for prescription drug users' fees as well as the products are entitled to a seven-year of market exclusivity and will not be affected by patents.
About NR082 (rAAV2-ND4; NFS-01 Project)
LHON disease is caused by mutations in mitochondrial DNA 11778, 14484 or 3460. ND4 gene of 11778 G>A mutation is the main pathological factor, which exists in 55-70% of European and American patients and 90% of Chinese patients. NR082 (NFS-01 project) is an innovative candidate drug for ophthalmic AAV-based gene therapy. It uses AAV2 vector to express human ND4 gene in the retinal ganglion cells to repair optic neuropathy caused by 11778 G>A mutation.
As early as 2011, Professor Bin Li's team started the world's first LHON gene therapy investigator-initiated trial (IIT) with this candidate drug. Nine subjects who participated in the clinical trial have been followed up for up to 8 years with no serious adverse reactions, and 5 ...
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