Pharma Focus America

DTx Pharma Granted FDA Orphan Drug Designation for DTx-1252 in the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)

Thursday, June 08, 2023

DTx Pharma, a biotechnology company focused on overcoming the challenges in delivering oligonucleotide therapeutics, has recently announced that its investigational FALCON small interfering RNA (siRNA) therapeutic, DTx-1252, has been granted Orphan Drug Designation by the United States Food and Drug Administration (FDA). The designation is specifically for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A), a progressive neuromuscular disorder that results in lifelong muscle function loss and disability.

Receiving Orphan Drug Designation is a significant milestone for DTx-1252 and highlights the urgent need for effective treatments for patients with CMT1A. Dr. Artie Suckow, the co-founder and CEO of DTx Pharma, expressed his satisfaction with the designation, emphasizing that DTx-1252 targets the underlying genetic cause of CMT1A and makes use of their FALCON platform to harness the potential of RNA interference (RNAi) therapeutics. The company is eager to continue advancing DTx-1252 in its development.

Orphan Drug Designation is granted by the FDA's Office of Orphan Products Development (OOPD) to drugs and biologics that demonstrate potential in treating, diagnosing, or preventing rare diseases affecting fewer than 200,000 individuals in the United States.

Notably, there are currently no approved therapeutics addressing the underlying genetic cause of CMT1A, which involves the overexpression of the PMP22 gene. This presents a significant unmet medical need for the estimated 150,000 patients living with this debilitating disease in the United States and Europe. DTx-1252, a novel and potential first-in-class candidate, specifically targets PMP22. The therapy has exhibited promising outcomes in preclinical studies, including the reversal of the disease in rodent models, successful translation to higher species, and nearing completion of IND-enabling studies.

CMT1A, as the most common inherited neuromuscular disease, has a profound impact on patients' lives. It leads to the overexpression of the PMP22 gene in Schwann cells, impeding peripheral nerve myelination and resulting in muscle wasting, neuropathic pain, mobility difficulties, and the inability to live independently.

DTx-1252 represents a potential breakthrough in addressing CMT1A by targeting the genetic cause of the disease. The Orphan Drug Designation granted to DTx-1252 offers hope for the patients affected by this condition, as it signifies recognition of the therapeutic's potential and provides incentives to facilitate its development and availability.

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