NMD Pharma Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease
- This is the 2nd orphan drug designation granted to the skeletal muscle-specific drug therapy NMD670
- Under this newly granted orphan drug designation, NMD670 is currently being evaluated in a Phase 2 clinical trial in adult patients with any genetically confirmed Type 1 or Type 2 CMT subtype
- NMD Pharma has three ongoing global clinical trials investigating NMD670 across rare neuromuscular diseases characterized by a high degree of patient impact and need, including a Phase 2 study in adults living with spinal muscular atrophy (SMA) type 3, a Phase 2b study in gMG patients, and this Phase 2 CMT study, with data readout(s) occurring from 2H/2025 through 1H/2026
Aarhus, Denmark, 6 January 2025 - NMD Pharma A/S, a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, today announces that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for NMD670, a novel, oral, small molecule inhibitor of the skeletal muscle-specific chloride ion channel ClC-1, for the treatment of Charcot-Marie-Tooth disease (CMT).
CMT is a hereditary neuropathy characterized by muscle weakness and fatigue with no approved therapies. An estimated 136,000 individuals in the US have CMT and it is estimated that over 3 million individuals worldwide live with CMT. Clinical features of this rare neuromuscular disease include motor signs and symptoms such as muscle weakness, muscle atrophy, and fatigue, as well as sensory deficits that, in combination, may cause substantial reduction in quality of life.
"NMD Pharma is committed to addressing the need of patients living with neuromuscular diseases such as CMT, and we are thrilled that the FDA has granted orphan drug designation to NMD670," commentedThomas Holm Pedersen, Chief Executive Officer of NMD Pharma. "Based on positive results from preclinical studies and the recently published ESTABLISH1CMT observational study, this designation not only highlights the urgent need for novel, effective treatments for this rare disease, but also underscores the therapeutic potential of our skeletal muscle-specific ClC-1 inhibitor approach to address the associated muscle weakness and fatigue."
In November 2024, NMD Pharma announced the initiation of a Phase 2 clinical trial, named SYNAPSE-CMT, of a twice daily oral dose of NMD670 over 21 days in 80 adult patients with any genetically confirmed CMT1 or CMT2 subtype, taking place across clinical sites in both the U.S. and Europe. Patients with CMT Type 1 and Type 2 in these geographies are encouraged to participate in the study, and for further information and a list of currently active trial sites, please look to clinicaltrials.gov under the identifier NCT06482437, or contact the Company at contact@nmdpharma.com.
In addition, NMD670 was previously granted ODD by the FDA for the treatment of generalized myasthenia gravis.
FDA ODD provides orphan status to therapies intended for the prevention, diagnosis or treatment of diseases affecting fewer than 200,000 people in the US.
1. Exploring SynapTic ABnormaLitIeS in Hereditary neuropathies
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Contacts
NMD Pharma A/S
Dan Brennan, SVP Corporate and Commercial Strategy
E-mail: contact@nmdpharma.com
ICR Healthcare
Mary-Jane Elliott / Ashley Tapp / Lindsey Neville
E-mail: NMDPharma@icrhealthcare.com
Tel: +44 (0)20 3709 5700
About NMD Pharma
NMD Pharma A/S is a clinical-stage biotech company developing a first-in-class platform of small molecule therapies selectively targeting the skeletal muscle chloride ion channel.
About NMD670
NMD670 is NMD Pharma's lead development program. It is a first-in-class small molecule inhibitor of the skeletal muscle specific chloride ion channel 1 (CIC-1). NMD Pharma has demonstrated that CIC-1 inhibition amplifies the muscle's responsiveness to weak signals, improving neuromuscular transmission and restores skeletal muscle function. This novel treatment approach has resulted in clinical evidence of CIC-1 inhibition in myasthenia gravis and preclinical evidence in spinal muscular atrophy, Charcot-Marie Tooth disease and sarcopenia. NMD670 has also been granted orphan-drug designation by the U.S. FDA for treatment of gMG.
About Charcot-Marie-Tooth disease (CMT)
CMT encompasses a group of hereditary sensory and motor neuropathies that cause damage to peripheral nerves and their neuromuscular junction. Damage caused by CMT worsens slowly over time and can result in substantial reductions in mobility, independence and quality of life due to muscle weakness, fatigue and atrophy across skeletal muscle groups such as the legs, feet, arms, hands and potentially leading to diaphragm weakness and paralysis. CMT is often broadly grouped into demyelinating (CMT type 1) and axonal (CMT type 2) based on nerve conduction studies, but there are more than 160 subtypes of CMT based on their genetic causes, clinical features, and progression patterns. CMT affects approximately 136,000 individuals in the United States and 3.2 million worldwide, with first symptoms typically appearing during adolescence or early adulthood.
