Actio Biosciences Receives Orphan Drug and Rare Pediatric Disease Designations for ABS-0871 for the Treatment of Charcot-Marie-Tooth Disease 2C

SAN DIEGO--(BUSINESS WIRE)--Actio Biosciences, a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced the U.S. Food and Drug Administration (FDA) has granted both orphan drug designation (ODD) and rare pediatric disease designation (RPDD) for ABS-0871, a TRPV4 inhibitor, for the treatment of TRPV4+ Charcot-Marie-Tooth disease subtype 2C (CMT2C).

“TRPV4 mutations cause CMT2C, a devastating disease that leads to debilitating and life-threatening symptoms, such as severe muscle weakness, vocal cord paresis and respiratory complications,” said David Goldstein, Ph.D., co-founder and CEO of Actio. “We are pleased to have received these designations from the FDA for ABS-0871 for CMT2C, a decision that underscores the recognition of the urgent need for new treatments for these patients.”

“We are thrilled to celebrate our STAR Alliance Partner, Actio Biosciences, for receiving both orphan drug and rare pediatric disease designations from the FDA for ABS-0871 in CMT2C,” said Charcot-Marie-Tooth Association CEO Sue Bruhn, Ph.D. “The collaborative efforts showcased through our Patients as Partners platform were instrumental in reaching this milestone, demonstrating the power of uniting the CMT community with industry leaders. This significant progress is a testament to the strength and dedication of our community, who made this achievement possible.”

The FDA grants orphan drug designation, also referred to as orphan status, to therapies intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. This designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved. Separately, rare pediatric disease designations are granted for rare diseases that primarily affect children under 18 years old with recipients of this designation being awarded a priority review voucher, upon approval. The priority review voucher may be redeemed, transferred, or sold.

About TRPV4 and ABS-0871

TRPV4 mutations cause two classes of rare autosomal dominant disorders: peripheral neuropathies including CMT2C, and skeletal dysplasias. Currently, there is no specific treatment available for TRPV4-mediated diseases.

Actio’s lead small molecule program, ABS-0871, targets TRPV4 mutations and is advancing towards the clinic for the treatment of CMT2C and TRPV4 related bone diseases. Evaluation of this program in novel construct-valid preclinical TRPV4 rare disease models has demonstrated marked improvements in motor function and mobility, compared to untreated controls.

About Actio Biosciences

Actio Biosciences is leveraging advances in precision medicine to develop new therapeutics that target shared genetics in rare and common diseases—bringing meaningful medicines from one to many. Applying its expertise in genetics, drug discovery and data sciences, Actio seeks to identify programs where both biological and technical risk can be minimized to streamline the drug development process and bring forward exceptionally potent and precisely targeted therapeutics. Founded in October 2021, the San Diego-based company is led by leaders in genetics and drug development and backed by top healthcare investors. For more information, please visit ActioBiosciences.com.