US FDA grants orphan drug status to mexiletine hydrochloride: Lupin

Topics Lupin | Drug major Lupin | USFDA

The company has already inked distribution agreements with three companies to make the drug commercially available in select European countries.

Pharma major Lupin on Monday said the US health regulator has granted orphan drug designation (ODD) to mexiletine hydrochloride for the treatment of myotonic disorders.

The Mumbai-based company's drug NaMuscula (mexiletine hydrochloride) is the first and only licensed product to treat myotonia in adults with non-dystrophic myotonic (NDM) disorders.

The company has already inked distribution agreements with three companies to make the drug commercially available in select European countries.

"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," Lupin CEO Vinita Gupta said.


The US Food and Drug Administration (US FDA) grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than two lakh people in the US.

Myotonic disorders are a group of heterogeneous, inherited, neuromuscular disorders characterised by a shared symptom called myotonia, which is an inability to relax a contraction of skeletal muscle.

The muscular contraction can take place while doing everyday activities like shaking someone's hand, blinking, walking across a street and climbing stairs.

Mexiletine hydrochloride reduces myotonia symptoms, resulting in a significant improvement in patient quality-of-life and other functional outcomes.

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