China CDE Grants Breakthrough Therapy Designation BTD to Neurophths NR082 in LHON

Neurophth Therapeutics, Inc., announced that the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has granted a Breakthrough Therapy Designation (BTD) to 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. NR082 (rAAV2-ND4), a recombinant adeno-associated viral vector, serotype 2, containing human ND4 codon-optimized 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. Phase I/II/III clinical trials are currently underway after the IND clearance by the China NMPA. Leveraging gene therapy strategy, NR082 viral vector is used through intravitreal injection to deliver the corrective genes to the patients' damaged retinal ganglion cell (RGC), therefore to repair the mitochondrial respiratory chain, improve the sensory function of the RGC and restore the visual function in LHON patients. A breakthrough therapy must provide effective treatment or prevention for a seriously debilitating or life-threatening condition that has no effective therapy or demonstrate substantial improvement over available therapies. Breakthrough Therapy Designation instituted in China is designed to expedite the development and review of innovative drugs presenting significant clinical advantages. According to the CDE, BTD provides opportunities for more intensive CDE guidance and discussion with respect to clinical trials and development strategy, and for priority review later. "The BTD is a great milestone for Neurophth and NR082. It's also an important advancement toward addressing the unmet medical need in LHON patients with a China gene therapy." said Prof. Bin Li, Founder, Chairman and CEO of Neurophth, "Further support from the CDE will help us accelerate the clinical development and commercialization of NR082. Neurophth will advance the Phase III clinical trial in China, as well as clinical trials in the U.S. Our team is fully committed to bringing NR082 to the market as soon as possible, alleviating patients from ophthalmic diseases with a transforming gene therapy." Ms. Yiyuan Chen, Head of Global Regulatory Affairs of Neurophth, commented, "We are very pleased to receive CDE's BTD, which is another good news following the orphan drug designation (ODD) by the U.S. FDA and EMA, and the IND clearance by both China NMPA and U.S. FDA. This reinforces our confidence in advancing NR082 to be the first marketed gene therapy for ophthalmic diseases in China. Neurophth will continue our work in hopes that this new gene therapy will soon benefit patients around the world."

Optimize Your trial insights with Clival Database.

Are you exhausted from the uncertainty of trial insights pricing? Clival Database ensures the clarity in the midst of the global scenario for clinical trials to you.

Clival Database is one of the best databases that offers an outstanding number of clinical trial data in terms of 50,000+ molecules and from primary regulatory markets as well as new entrants like Indian and Chinese markets.

With Clival, you get accurate positioning of historical sales data, patent database, company profiling, safety & efficacy, and prediction of launch of new innovative molecules helping you to align your research and driving down the cost.

To add value, we further break down our analytics for you so that improving your operational effectiveness; optimizing your clinical trials; and offering you accurate and high-quality data at lowest possible prices becomes possible.

Elevate your trial success rate with the cutting-edge insights from Clival database.

Check it out today and make more informed sourcing decisions! Learn More!