Neurophth Therapeutics' Treatment of Leber's Hereditary Optic Neuropathy Gene Therapy NR082 Granted Orphan Drug Designation by EMA

Neurophth Therapeutics announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) granted the orphan drug designation (ODD) for the company's leading gene therapy drug candidate, NR082 (rAAV2-ND4), for the treatment of Leber's hereditary optic neuropathy (LHON) associated with mtND4 mutation. 

"The positive opinion from the COMP acknowledges the compelling IND-enabling data and clinical data of 186 subjects from three investigator-initiated trials (IITs)," Dr. Bin Li, Founder of Neurophth, said in a company news release. "Some of the subjects in the earlier IIT were followed for up to 75-90 months, demonstrating excellent clinical durability. Our integrated phase 1/2/3 registrational trial is ongoing, and the data from this trial are intended to support global regulatory filings."

"This orphan drug designation is an important milestone toward addressing the unmet need of families and patients living with LHON, and it follows the ODD previously granted in 2020 by the US. Food and Drug Administration (FDA) for this same gene therapy drug candidate as a potential treatment for mtND4-LHON," said Xin Zhang, MD, MSc, Chief Medical Officer and Chief Operations Officer at Neurophth. "In China, for Global - as Neurophth expands its regulatory strategy in Europe, we will continue to make meaningful changes in the lives of patients with genetic diseases around the world."

Ms. Yiyuan Chen, Head of Global Regulatory Affairs of Neurophth, commented, "We are very pleased to receive EMA's orphan drug designation, which is another good news following the recent US IND clearance of NR082 and the grant of US Orphan Drug Designation of NFS-02. This is a true reflection of the company's operational excellence. We will continue to deliver our promise to bring transformative treatments to patients around the world."

About Orphan Drug Designation

EMA orphan designation is designed to encourage the development of new treatments to treat a seriously debilitating or life-threatening condition that affects fewer than 5 in 10,000 people in the European Union (EU), and there must be sufficient non-clinical or clinical data to suggest the investigational medicine may produce clinically relevant outcomes. Medicines that meet the EMA's orphan designation criteria qualify for several incentives, including 10 years of market exclusivity, clinical protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU member states, and potentially reduced fees for regulatory activities. Applications for orphan designation are examined by the EMA's Committee for Orphan Medicinal Products (COMP), using the Committee's network of experts.

About NR082

Investigational NR082 (rAAV2-ND4), a novel recombinant adeno-associated viral vector, serotype 2, containing a mitochondria codon-optimized NADH-dehydrogenase subunit 4 (ND4) gene under the control of the cytomegalovirus promoter and enhancer, is a novel gene therapy product that is being developed for the treatment of Leber hereditary optic neuropathy (LHON) associated with mtND4 mutations. The U.S. Food and Drug Administration (FDA) granted orphan-disease designation to NR082 in September 2020^[6]. Safety and efficacy of mtND4 gene therapy have been evaluated in three investigator-initiated trials (IITs) with clinical durability up to 90 months in the first IIT. The results of these three IITs of 186 LHON patients demonstrated that an intravitreal injection of rAAV2-ND4 in subjects with LHON is well tolerated and can be effective at improving visual acuity^[7,8,9].