Here's the news about NeuroVenti

Fragile X Syndrome Treatment Pipeline' Approved by the U.S. FDA for Or…

페이지 정보

조회 69회 작성일 22-10-26


The only company in Korea specialized in the development of therapies for rare brain developmental disorders

Preparing for a phase 2 clinical trial for autism spectrum disorder in Korea in 2023

Selected to present at Bio Europe 2022 and is currently seeking partnerships with multiple global pharmaceutical companies

On October 24, 2022, NeuroVenti, Inc. announced that it had obtained orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for its candidate drug for fragile X syndrome (FXS), a rare genetic disorder that causes developmental problems. 

The ODD program is designed to promote the development of therapies for rare diseases by providing various incentives for development and sales through the certification of substances that can be used to treat intractable rare diseases.


The main incentives for rare disease drug development include a 7-year marketing exclusivity for approved drugs, a 25% tax deduction for clinical research costs in the United States, a waiver of new drug approval fees under the Prescription Drug User Fee Act (PDUFA) for orphan drugs, and support for drug development meetings with the FDA Office of Orphan Products Development, including the exclusion of orphan product reviewers. Many pharmaceutical companies are pursuing rare disease designation for their drugs and drug candidates to receive benefits such as research grants from the US Orphan Products Development Program (OOPD) to support rare disease clinical trials.


Fragile X syndrome (FXS), a rare disease recently designated as an orphan drug, is a genetic developmental disorder caused by a lack of FMRP protein, which manifests various neuropsychiatric symptoms such as social absence, hyperactivity, impulsivity, compulsive behavior, and intellectual disability. The prevalence rate is 0.03%, and it affects more males than females due to X chromosome inheritance. There is currently no approved treatment worldwide, and it represents a significant unmet medical need. As severe autism symptoms often accompany FXS, researchers are looking for solutions through autism research and drug development.


NeuroVenti, Inc. is a domestic bioventure founded by Professor Shin Chan Young of the Department of Pharmacology at Kunkuk University College of Medicine in 2015, specializing in developing treatments for rare brain developmental disorders such as FXS, autism spectrum disorder, ADHD, and tic disorders. They are currently preparing to conduct Phase 2 clinical trials for autism spectrum disorder in Korea in 2023, and have been selected as a presenting company at Bio Europe 2022 (October 24-26, 2022), where they will attempt to collaborate and partner with numerous global pharmaceutical companies.


Through this rare disease designation, it is expected that the development of treatments for autism, including FXS, will receive a boost in technology transfer, clinical entry, and approval processes.

Reporter 이석훈