RESTEM, a clinical-stage biotechnology company, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its umbilical cord outer lining stem cells (ULSCs) program for the treatment of Polymyositis (PM) and Dermatomyositis (DM). This designation aims to support the development of a novel therapy for these rare autoimmune diseases.
Polymyositis and dermatomyositis are autoimmune diseases characterized by inflammation and weakness of the skeletal muscles. Dermatomyositis also presents with a distinctive skin rash. These conditions can severely impact a patient's quality of life and pose significant treatment challenges. The estimated incidence of PM/DM ranges from 5 to 22 cases per 100,000 individuals.
Clinical Significance of ULSC Therapy
Current treatments for PM/DM primarily involve long-term use of corticosteroids and immunosuppressive therapies, which are often associated with substantial toxicity and adverse side effects. RESTEM's ULSC therapy offers a potential alternative by modulating the immune system, aiming to reduce the need for steroids.
"Receiving the Orphan Drug Designation (ODD) is a significant milestone in our mission to provide effective and safer treatment options for patients suffering from PM/DM," said Keith March, MD, PhD, Chief Medical Officer of RESTEM. "We believe that our novel ULSC therapy has the potential to offer meaningful benefits for patients by modulating the immune system, which may reduce the reliance on steroids. We look forward to the continued development of the ULSC platform and working closely with regulators to bring its potential to patients in need."
Ongoing Clinical Development
RESTEM's ULSC therapy has shown promising results in early clinical trials, demonstrating safety, tolerability, and initial clinically significant improvements. These trials also suggest the potential to significantly reduce the need for steroid use in patients. The company is currently preparing for Phase 2/3 trials, with anticipated initiation in the first quarter of 2025.
Benefits of Orphan Drug Designation
The FDA's Office of Orphan Products Development grants ODD status to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. The designation provides several benefits to drug developers, including assistance in the drug development process, tax credits for qualified clinical costs, exemptions from certain FDA fees, and seven years of marketing exclusivity upon approval.
