(CARY, N.C.) — NEWS: Rescindo Therapeutics Inc., a drug discovery company dedicated to the development of new treatments for rare genetic disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation and Rare Pediatric Disease Designation for the Company’s lead drug RSC-57, for the treatment of patients with Kabuki Syndrome.
“We are honored to receive these very important designations for our lead drug for Kabuki Syndrome. The decision validated the urgency and need for treatment of this devastating disease for which there are no FDA-approved therapies,” said Dr. Athanasios (Thanos) Maroglou, co-founder, President and CEO of Rescindo. “With RSC-57 being a repurposed drug with a large established safety profile, we hope to proceed with clinical trials rapidly after the completion of our pre-IND package and guidance from the FDA.”
Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity.
Rare Pediatric Disease Designation is granted by the FDA in the case of serious or life-threatening diseases affecting fewer than 200,000 people in the United States, primarily those 18 years of age and younger. The sponsor of a drug with Rare Pediatric Disease Designation may, upon marketing approval, qualify for receipt of a priority review voucher applicable to a subsequent marketing application, which voucher is fully transferable.
About Kabuki Syndrome
Kabuki Syndrome is a rare developmental disorder characterized by intellectual disability, muscle hypotonia, distinctive facial features, short stature, immune deficiency and malformations of the cardiac, renal, gastrointestinal, and/or skeletal systems. Typically diagnosed during early childhood, it is possible to identify KS as early as infancy based on characteristic facial features and associated organ malformations.
The global prevalence of KS is estimated at 1:32,000 births, although its incidence is likely underestimated, as judged by the acceleration of new cases now that genome sequencing is becoming more broadly available. At present, KS has no cure.
About Rescindo Therapeutics Inc.
Rescindo Therapeutics is drug discovery and development company focused on rare genetic diseases to improve peoples’ lives.
Rescindo Therapeutics Inc. was founded in 2015 by Dr. Athanasios Maroglou and Dr. Nicholas Katsanis with an objective to discover and develop new drugs for children with rare genetic diseases. Using a novel approach that combines genetics, molecular biology, and high throughput use of model organisms, we accelerate the discovery of new therapies. Together with our academic, private sector partners, and patient advocacy groups, we are working toward bringing new treatments for life-threatening disorders.
Learn more at: https://www.rescindotherapeutics.com/
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