siRNA therapy for rare disease granted orphan status


The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to DTx Pharma’s DTx-1252, an investigational small interfering RNA (siRNA) therapeutic for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A).

CMT1A is a progressive, neuromuscular, autosomal-dominant disease that leads to life-long loss of muscle function and disability.

“We are pleased to receive Orphan Drug Designation from the FDA for DTx-1252. Patients living with CMT1A are in urgent need of treatment and this represents a significant step forward for the patients and the DTx team as we advance DTx-1252 toward the clinic,” said Artie Suckow, Co-Founder and CEO of DTx Pharma.

“DTx-1252 targets the underlying genetic lesion of the disease and leverages our Fatty Acid Ligand Conjugated OligoNucleotide (FALCON) platform to unlock the promise of RNAi therapeutics. We look forward to the continued development of DTx-1252.”

The FDA Office of Orphan Products Development (OOPD) provides orphan status to drugs and biologics which are intended to treat, diagnose or prevent rare diseases that affect fewer than 200,000 people in the US.

Currently, there are no approved therapeutics addressing PMP22, the underlying genetic cause of CMT1A, for the estimated 150,000 patients living with the debilitating disease in the US. and Europe.

DTx-1252 is a novel, potential first-in-class candidate targeting PMP22. The asset boasts a robust preclinical package, demonstrating reversal of disease in preclinical rodent models and translation to higher species with IND-enabling studies near completion.

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