Spain approves reimbursement of Lupin NaMuscla for symptomatic treatment of myotonia in adults with non-dystrophic myotonic disorders

NDM disorders are a group of rare, inherited neuromuscular disorders with an estimated prevalence of 1 case per 100,000 inhabitants in Spain, and is characterized by the inability to relax muscles following voluntary contraction (myotonia). NaMuscla reduces myotonia symptoms in people with NDM, resulting in a significant improvement in quality of life and other functional and clinical outcomes for patients. NaMuscla, which has been designated orphan drug status, received EU marketing authorization in December 2018.

“NDM patients in Spain will now be able to access NaMuscla in a sustainable way. We are grateful to all stakeholders involved in the approval process, especially our partner Exeltis,” said Thierry Volle, President EMEA, Lupin. “Lupin’s transformational journey into specialty disease areas continues, with future investments planned for neuromuscular diseases, to meet the unmet needs of patients.”

“We are excited by this decision which will enable us to provide a new, innovative treatment option that addresses the unmet needs of patients with NDM as soon as possible,” said Alberto Fábregas Gil, Director General Spain and Portugal, Exeltis. “This news allows us to distribute NaMuscla quickly and effectively and strengthen our position in the central nervous system area.”

To date, people in Spain living with NDM have had limited access to a licensed treatment for myotonia that can reduce the daily burden of this disabling, lifelong condition. Limited access leads to inconsistent medication supply, administrative challenges, and associated financial burdens. This, alongside limited clinical experience among healthcare professionals due to the rare nature of the disease, may result in significant harm to patients.

Lupin’s pediatric trial (NCT04624750), part of the pediatric investigation plan for NaMuscla in children with myotonic disorders, is ongoing and successfully concluded patient enrolment in a first patient cohort group who were offered and rolled over into a 2-year follow-up study (NCT04622553). A post-authorization study to address long-term safety and treatment effects of NaMuscla on patient-reported outcomes in adults with NDM (NCT04616807) has concluded patient enrollment and will provide 3 years prospective data on NaMuscla in a real-life setting.