Zug, Switzerland—Today Lupin Limited announced that NaMuscla (mexiletine) has been launched in Germany and the United Kingdom (UK). NaMuscla is approved across the European Union (EU) for the symptomatic treatment of myotonia in adults with non-dystrophic myotonic (NDM) disorders.
NaMuscla is an antimyotonic agent licensed to treat symptomatic myotonia in adults with non-dystrophic myotonic disorders. In randomized controlled trials, NaMuscla (167 to 500 mg mexiletine/day) has been shown to significantly reduce myotonia compared to placebo, restoring skeletal muscle hyperexcitability through its use-dependent, voltage-gated, sodium channel-blocking actions which are independent of the cause of channel function. This resulted in an improvement in patient quality-of-life and other functional outcomes. Gastrointestinal discomfort was reported as the most common adverse event, demonstrating NaMuscla to be safe and generally well tolerated.
The launch of NaMuscla in Germany and the UK follows the European Commission’s approval of the product on December 18th, 2018. NaMuscla, designated an orphan drug by the European Medicines Agency (EMA), is the first treatment to be licensed across the EU for the symptomatic treatment of myotonia in adults with NDM disorders. The product will be commercialized in Germany by Hormosan Pharma GmbH, a full subsidiary of Lupin Ltd., and in the UK by Lupin Healthcare (UK) Ltd.
“We are delighted to be able to provide patient and market access to NaMuscla in Germany and the UK,” said Thierry Volle, President EMEA, Lupin. “NaMuscla is the first licensed therapy for myotonia across the EU and offers an effective treatment option for patients living with this life-altering symptom. We look forward to additional launches for patients in other EU territories through 2019 and 2020.”
NDM are a subset of rare, inherited myotonic disorders which are caused by mutations within ion channels in the sarcolemma membrane of skeletal muscles. Non-dystrophic myotonias exhibit both sodium and chloride channelopathies, which result in altered membrane excitability. For patients with NDM, myotonia is the most prominent symptom and demonstrates different phenotypes in subgroups of NDM disorders. It can affect different parts of the body, such as legs, arms or facial muscles, more severely.
Myotonia in NDM patients has an onset in childhood and persists over the patient’s lifetime – significantly impacting the patient’s daily life. Myotonia is described by patients in a variety of ways (stiffness, cramps, pain, difficulty releasing a fist, or difficulty swallowing or eating) which can contribute to substantial delays in diagnosis and treatment, leading to decreased patient quality of life and often significant disability.
Prior to the approval of NaMuscla, the vast majority of more than 7500 people in Europe living with NDM had limited access to a licensed treatment for myotonia which reduced symptoms. Limited access leads to inconsistent medication supply, administrative challenges to both treating physicians and patients and associated financial burdens. The EMA recognized the importance of having an effective therapy in adult NDM patients throughout the EU by granting NaMuscla orphan drug status.
Lupin has ongoing partnering discussions for the commercialization of NaMuscla in European territories outside of Germany and the UK.