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news.The US Food and Drug Administration (FDA) has granted orphan drug designation and rare pediatric disease designation to ARMGO Pharma's ARM210 for the treatment of duchenne muscular dystrophy (DMD).
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DMD is a rare form of muscular dystrophy that leads to progressive and debilitating weakening of muscles in young males.
It occurs due to mutations in the gene that makes dystrophin, a protein essential for the normal structure and function of skeletal and cardiac muscles.
ARM210, also known as S48168, targets the ryanodine receptor (RyR), an intracellular calcium release channel that becomes leaky in disease states including DMD, resulting in muscle damage and loss of function.
The phase 1 clinical trial for ARM210 has started in Europe. It will assess its safety and pharmacokinetics in healthy male subjects.
ARMGO aims to start a Phase 2a study on subjects with DMD after the successful completion of the Phase 1 trial.
ARMGO Pharma president and CEO Sapan Shah said: "These designations from FDA represent an important achievement for the ARM210 program and highlight the ongoing need to provide meaningful treatments for patients and families affected by Duchenne Muscular Dystrophy.
"We are looking forward to continuing our progress with the ARM210 clinical program, including completing ongoing Phase 1 clinical studies and advancing into Phase 2 studies in DMD patients."