RESTEM, a biotechnological firm at the clinical-stage, dedicated to crafting innovative cell therapies that are readily available and aim to regulate the immune response, has disclosed that the United States Food and Drug Administration (FDA) has bestowed Orphan Drug Designation (ODD) upon its program involving stem cells from the umbilical cord’s exterior lining, targeting polymyositis (PM) and dermatomyositis (DM) treatment.
Polymyositis is an autoimmune disorder that results in inflammation and weakness affecting the skeletal muscles crucial for movement. Dermatomyositis is a variant of polymyositis, additionally presenting with a skin rash along with muscle inflammation. Both conditions severely affect the quality of life and pose significant treatment challenges. The occurrence rate of PM/DM ranges from about 5 to 22 cases per 100,000 people. Existing treatment methods often involve the prolonged use of corticosteroids alongside immunosuppressive medications, which may lead to substantial side effects and toxicity.
“Our achievement of receiving Orphan Drug Designation (ODD) aligns perfectly with our commitment to offer enhanced and safer treatment alternatives for those battling PM/DM,” expressed Keith March, MD, PhD, the chief medical officer at RESTEM. “Our innovative ULSC therapy is poised to provide critical advantages for patients by potentially minimizing dependency on steroids through immune system modulation. We anticipate progressing our ULSC platform and collaborating closely with regulatory authorities, aiming to fulfill patients’ needs.”
According to previous disclosures, RESTEM’s ULSC therapy has exhibited encouraging outcomes in early-stage clinical trials, highlighting safety, tolerance, and noteworthy early clinical improvement potential, alongside the possibility of drastically reducing steroid utilization in patients. The company is in the process of setting up phase 2/3 trials, projected to commence in the first quarter of 2025.
The FDA’s Office of Orphan Products Development assigns Orphan Drug Designation to pharmaceuticals and biologics aimed at the safe, effective treatment, diagnosis, or prevention of rare ailments or conditions affecting under 200,000 individuals across the U.S. The designation offers several incentives to developers to facilitate the development of drugs and biologics for patient groups with unmet medical needs, including development process assistance, tax breaks on qualifying clinical costs, exemptions from certain FDA fees, and marketing exclusivity spanning seven years.
RESTEM stands at the forefront as a clinical-stage biotechnology enterprise, centered on the creation of advanced, readily available cell therapies designed to modulate the immune system effectively.