COLUMBUS, October 2023 – Armatus Bio, a late-preclinical stage biotech innovator developing vectorized RNAi medicines in neuromuscular disorders, has received Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) designation from the U.S. Food and Drug Administration (FDA) for TVR110, an advanced engineered miRNA under development for Charcot-Marie-Tooth disease type 1A (CMT1A), a progressively debilitating disorder with no approved treatments.
“Our receipt of ODD and RPD from the FDA highlights the overwhelming need for therapeutic innovation to support people living with CMT1A in the U.S.,” said Rachel Salzman, DVM, Chief Executive Officer of Armatus Bio. “We continue to build momentum with TVR110 based on robust preclinical signals, and are now actively advancing this asset toward the clinic.”
TVR110 is an AAV-delivered microRNA that safely reduces PMP22 overexpression, which is the underlying cause of CMT1A. Preclinical evaluations have demonstrated encouraging findings that the vectorized RNAi strategy can bring PMP22 down to healthy levels, with signs of improvement in nerve histology, repaired myelin and restored functional behavior.
ODD is granted to therapeutic candidates for conditions affecting fewer than 200,000 people in the U.S. This designation provides incentives to advance clinical development with potential for up to seven years of U.S. market exclusivity if the product is approved for its designated indication. RPD is available for therapeutics targeting diseases that primarily affect children ages 18 or younger and fewer than 200,000 people in the U.S. Once awarded, these vouchers may be transferred or sold to other companies.
About CMT1A
Charcot-Marie-Tooth disease type 1A is a peripheral nerve demyelination and axon degeneration disease caused by a duplication of the PMP22 gene, which leads to the production of an abnormal amount of peripheral myelin protein 22 (PMP22). The disease affects over 150,000 people in the U.S. and EU. It typically presents in the teenage or young adult years, and leads to progressive muscle weakness and sensory loss in the extremities that often results in severe disability, pain, and loss of independence. There are currently no approved disease-modifying therapies or curative interventions for CMT1A.
About Armatus
Armatus Bio is a late-preclinical stage, privately held biotech innovator developing advanced medicines that leverage vectorized RNAi (RNA interference). Armatus’ uniquely specific, engineered microRNAs are noncoding RNAs responsible for regulating gene expression by mirroring innate cellular biogenesis processes without altering the underlying genetic make-up. The company’s two lead assets are designed to target neuromuscular disorders: TVR110 for Charcot-Marie-Tooth disease type 1A (CMT1A), and ARM-201 for Facioscapulohumeral Muscular Dystrophy (FSHD), which together affect more than 225,000 people in the U.S. and European Union. In preclinical studies, these investigational drugs demonstrated robust signals of target engagement and biomarker improvement, and both are advancing toward the clinic. For more information, visit www.ArmatusBio.com.