'Fragile X Syndrome Treatment Pipeline' Approved by the U.S. FDA for Orphan Drug Designation

22-10-26

The only company in Korea specializing in the development of drugs for intractable brain development disorders

Currently preparing for domestic clinical phase 2 for autism spectrum disorder in 2023

Selected as Bio Europe 2022 presentation company Attempting cooperation and partnering with a number of global pharmaceutical companies

Neuroventi Co., Ltd. (CEO Shin Chan-young), which provides integrated solutions for intractable brain diseases, especially brain development disorders, received orphan drug designation from the U.S. Food and Drug Administration (FDA) on October 24, 2022 local time. ODD) announced on the 27th.

A treatment candidate for autism and fragile X syndrome (FXS) being developed by Neuroventi Co., Ltd. has been designated as an orphan drug designation (ODD) by the U.S. Drug Administration (FDA).

The Orphan Drug Designation (ODD) system aims to promote the development of rare disease treatments by providing various incentives for development and sales through the certification of substances that can be used to treat intractable rare diseases.

Key incentives include 7-year marketing exclusivity on approved drugs, a 25% tax credit on the cost of conducting clinical studies in the United States, and new drug approval review under the Prescription Drug User Fee Act (PDUFA) for orphan drugs. Administrative and financial support, such as fee waivers and the presence of an examiner from the FDA Office of Orphan Products Development at all meetings with the FDA related to drug development.

In addition, many pharmaceutical companies are challenging the orphan drug designation of their drugs and drug development candidates as they can receive benefits such as research subsidies from the Office of Orphan Drug Development (OOPD) to support clinical research on orphan drugs.

Fragile X syndrome (FXS), an incurable neurodevelopmental disorder that has been designated as an orphan drug this time, is a hereditary developmental disorder caused by a deficiency of FMRP protein. ∙It shows neurological symptoms, and the prevalence rate is 0.03%.

It is a disease caused by the X chromosome and is known to be more common in males than females, and there is no globally approved treatment so far, so it is a disease with great unmet medical demand. In many cases, severe autism symptoms are displayed, so solutions are being sought through autism research and treatment development.

Neuroventi is a bio venture founded in 2015 by Professor Shin Chan-young of the Department of Pharmacology at Konkuk University College of Medicine.

We are currently preparing for phase 2 clinical trials for autism spectrum disorder in Korea in 2023, and have been selected as a presentation company for Bio Europe 2022 (October 24, 2022 - October 26, 2022) and are attempting to cooperate with a number of global pharmaceutical companies. We are partnering with

Through this designation as an orphan drug, it is expected to gain momentum in the development of autism treatments, including FXS, through technology transfer, clinical entry, and licensing processes.