US FDA grants Lupin’s Mexiletine Orphan Drug Designation

Pharma major Lupin announced today that the US Food and Drug Administration (US FDA) has granted Orphan Drug Designation (ODD) to mexiletine hydrochloride for the treatment of myotonic disorders.

Myotonic disorders are a group of heterogeneous, inherited, neuromuscular disorders characterized by a shared symptom called myotonia. Myotonia is an inability to relax a contraction of skeletal muscle which originates from a voluntary muscular contraction such as shaking someone’s hand and blinking, or everyday activities such as walking across a street and climbing stairs. Mexiletine reduces myotonia symptoms, resulting in a significant improvement in patient quality-of-life and other functional outcomes.

The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.

Vinita Gupta, CEO, Lupin said, “There is a serious unmet medical need for the management of symptoms in patients with myotonic disorders. The decision by the US FDA to grant orphan drug designation to mexiletine brings us closer to providing a licensed treatment option for patients in the US, and we are pleased to have this opportunity to further our commitment to these patients.”