The Charcot-Marie-Tooth Association (CMTA) is pleased to share exciting news from our CMTA-STAR Alliance Partner, Actio Biosciences. The company has received both orphan drug designation (ODD) and rare pediatric disease designation (RPDD) designation from the FDA for ABS-0871, a TRPV4 inhibitor for treating CMT2C. This achievement represents a crucial step forward for CMT2C patients and the CMT community.
“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 CMTA CEO Sue Bruhn, PhD. “The collaborative efforts showcased through our Patients as Partners in Research 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, reduced 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.
Actio Biosciences partnered with CMTA, leveraging our Patients as Partners in Research platform to connect with the CMT2C community and gain valuable patient perspectives.
“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 celebrate this encouraging news from Actio Biosciences and look forward to the continued progress in bringing effective treatments to those affected by CMT2C. Join us in supporting and celebrating this significant achievement as we work together towards a world without CMT.
Published: August 8, 2024
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