US FDA Grants Mexiletine Orphan Drug Designation

US FDA Grants Mexiletine Orphan Drug Designation

Zug, Switzerland, 8 June 2019: Lupin is pleased to announce that the US Food and Drug Administration (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 outcomes¹.

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 of 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.”

For further information or queries please contact:

Consilium Strategic Communications
Amber Fennell / Sukaina Virji / Lizzie Seeley
Tel: [phone]
Email: [e-mail]

Notes for Editors

About Myotonic Disorders
Myotonic disorders are a group of heterogeneous, inherited, neuromuscular disorders characterized by a shared symptom called myotonia. Myotonia is described as 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. Taken together, these myotonic disorders are rare and comprise two groups: the myotonic dystrophies and the non-dystrophic myotonic disorders³.

Myotonic dystrophy (DM) affects multiple systems in the body. The two types of DM (DM1 and DM2) differ in prevalence, affected genes, disease progression, symptoms and severity of symptoms, with sub classifications based on disease onset, making it difficult to know how the disorder will affect patients. DM1 is the most common form, with the global consensus that myotonic dystrophies, based upon estimates from the NIH and others for western European and North American populations, affect 12.5 per 100,000 individuals^4,5.