Cell and gene therapy company ENCell said on the 6th that its allogeneic umbilical cord-derived mesenchymal stem cell therapy candidate "EN001" received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for Duchenne muscular dystrophy (DMD), following Charcot-Marie-Tooth disease (CMT), a rare neurological disorder.
The FDA's orphan drug designation system is designed to encourage the development of new drugs for diseases with small patient populations. Once designated, developers can receive benefits such as seven years of market exclusivity after approval, tax credits for clinical expense, and expedited review.
The company said that with this designation, EN001 has been recognized for its treatment potential across two distinct rare disease areas—peripheral nerve disease (CMT) and degenerative muscle disease (DMD)—and that this demonstrates its potential as a platform therapy that can cover rare neuro-muscular diseases more broadly.
Duchenne muscular dystrophy is a fatal pediatric rare disease caused by a deficiency in the dystrophin gene, which produces a protein essential for maintaining muscle. As muscle strength progressively weakens, most patients lose the ability to walk after their teenage years. To date, treatment options that fundamentally halt disease progression remain limited. This disease shows progressive muscle degeneration, calling for treatment strategies that control inflammation in muscle tissue and preserve function.
EN001 is a therapy based on allogeneic umbilical cord-derived mesenchymal stem cells (WJ-MSC). It applies ENCell's proprietary cell-culture platform technology "ENCT (ENCell Technology)" to suppress cellular aging and enhance the secretion of anti-inflammatory and tissue-regeneration factors. The company believes this mechanism of action could be effective not only in repairing peripheral nerve damage but also in controlling inflammation in muscle tissue and preserving function.
Chief Executive Jang Jong-uk of ENCell said, "This additional orphan drug designation is an objective recognition that EN001 is a platform technology applicable across intractable rare diseases with high unmet medical needs, beyond a single indication," adding, "Securing ODDs in two indications has greatly expanded our options for global technology transfer and business expansion." Jang added, "We will accelerate clinical development and focus our capabilities on delivering tangible global results."