Actio Biosciences Gets Orphan Drug and Rare Pediatric Disease Status for ABS-0871 to Treat Charcot-Marie-Tooth Disease 2C

Actio Biosciences, a biotechnology firm focused on a pioneering approach to genetics and precision medicine, has announced that their TRPV4 inhibitor, ABS-0871, has received both orphan drug designation (ODD) and rare pediatric disease designation (RPDD) from the U.S. Food and Drug Administration (FDA). These designations are aimed at treating TRPV4+ Charcot-Marie-Tooth disease subtype 2C (CMT2C), a genetic disorder.

Dr. David Goldstein, co-founder and CEO of Actio Biosciences, highlighted the severe impact of CMT2C, which results in symptoms such as significant muscle weakness, vocal cord paresis, and respiratory issues. He expressed satisfaction with the FDA's recognition, underscoring the urgent need for new treatments for CMT2C patients.

The Charcot-Marie-Tooth Association's CEO, Sue Bruhn, Ph.D., praised Actio Biosciences for achieving these designations. She noted that the collaborative efforts made through the Patients as Partners platform played a crucial role in this accomplishment. This partnership underscores the collective strength and commitment of the CMT community and industry stakeholders, marking a significant milestone in the development of treatments for CMT2C.

The FDA grants orphan drug designation to therapies targeting rare diseases affecting fewer than 200,000 people in the U.S. This status provides several benefits, including tax credits for clinical testing, reduced FDA application fees, and seven years of market exclusivity upon approval. Rare pediatric disease designations are granted for conditions that primarily affect children under 18, offering a priority review voucher that can be redeemed, transferred, or sold upon approval.

TRPV4 mutations are responsible for rare autosomal dominant disorders, including peripheral neuropathies like CMT2C and skeletal dysplasias. Currently, no specific treatments are available for diseases mediated by TRPV4 mutations. Actio Biosciences’ leading small molecule program, ABS-0871, is designed to target these mutations. The program is advancing towards clinical trials for the treatment of CMT2C and TRPV4-related bone diseases. Preclinical models have shown significant improvements in motor function and mobility when treated with ABS-0871 compared to untreated controls.

Actio Biosciences is harnessing advancements in precision medicine to develop therapeutics targeting shared genetic factors in rare and common diseases. The company aims to streamline the drug development process by identifying programs where both biological and technical risks are minimized. Founded in October 2021 and headquartered in San Diego, the company is led by experts in genetics and drug development, supported by leading healthcare investors.

Actio Biosciences is dedicated to bringing effective and precisely targeted medicines to market, leveraging their expertise in genetics, drug discovery, and data sciences. Their approach aims to make a significant impact in the field of precision medicine, benefiting a wide range of patients with both rare and common genetic conditions.

In conclusion, the FDA’s ODD and RPDD designations for ABS-0871 represent critical steps forward in addressing the needs of patients with TRPV4+ CMT2C. The collaborative efforts and scientific advancements demonstrated by Actio Biosciences provide hope for effective treatments for this rare and challenging disease.