FDA Grants Rare Pediatric Disease and Orphan Drug Designations to Rescindo’s Treatment for Kabuki Syndrome

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designations to Rescindo Therapeutics’ lead drug RSC-57, for the treatment of patients with Kabuki syndrome.

Kabuki syndrome (KS) is a rare genetic 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 in 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.

Rescindo Therapeutics aims 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 to accelerate the discovery of new therapies.

RSC-57 is a repurposed small molecule drug with a large established safety profile.  and Rescindo hopes to proceed with clinical trials rapidly after the completion of its pre-IND package and guidance from the FDA.

“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 Athanasios Maroglou, co-founder, president and CEO of Rescindo.

The FDA grants Orphan Drug designation to drugs or biologics intended to treat a rare disease or condition 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.

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The designation makes each RSC-57 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Rhythm Pharmaceutics sold its priority review voucher for $100 million in January 2021 to Alexion Pharmaceuticals.

Photo: Athanasios Maroglou, co-founder, president and CEO of Rescindo