Lupin responds to NICE Appraisal Consultation Document (ACD) on NaMuscla (mexiletine) to treat symptomatic myotonia in adults with non-dystrophic myotonic disorders (NDM)

Lupin responds to NICE Appraisal Consultation Document (ACD) on NaMuscla (mexiletine) to treat symptomatic myotonia in adults with non-dystrophic myotonic disorders (NDM)

Slough, UK, 16th February 2021: Lupin is disappointed that the National Institute for Health and Care Excellence (NICE) has published an Appraisal Consultation Document (ACD)¹ recommending not to approve NaMuscla (mexiletine) for routine funding to treat symptomatic myotonia in adults with non-dystrophic myotonic disorders (NDM) in England and Wales.

The ACD document is a preliminary recommendation at this stage and may change after further consultation. Lupin is committed to working with NICE to ensure patients in England and Wales have access to the first and only licensed medication for NDM. The Scottish Medicine’s Consortium approved the funding of Namuscla in December 2020 through its orphan drug PACE process. Lupin believes patients living with symptomatic NDM have the right to licensed treatment choices that improve their lives.

NDM is an ultra-rare genetic neuromuscular disorder, affecting 300-400 patients in England and Wales, in which the sufferer's muscles are slow to relax after movement. These symptoms occur intermittently and unexpectedly, often causing muscle weakness, pain, fatigue and impairment of physical activities.²

The European Commission (EC) approved NaMuscla for the symptomatic treatment of myotonia in adults with non-dystrophic myotonic (NDM) disorders in January 2019. NaMuscla has received orphan drug designation from the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP).³

Ben Ellis, Lupin UK General Manager, said: “We are concerned for NDM patients in England and Wales who are currently stable on NaMuscla. In the event of a negative NICE outcome, these patients may be moved to an unlicensed therapy as an alternative, or have no medication. We will work together with NICE to try and find a resolution to prevent this, with equity of patient access across the whole of the UK being our main priority. NaMuscla is the standard of care for patients with NDM, an ultra-rare disease. We are disappointed that the assessment was not assigned as a Highly Specialised Technology appraisal, which was designed to better suit ultra-rare disease technologies such as NaMuscla.”