Scottish Medicines Consortium approves NaMuscla(R) (mexiletine) to treat 
symptomatic myotonia in adults with non-dystrophic myotonic disorders 
(NDM) 
 
   Slough, UK, 8 December 2020: Lupin welcomes the Scottish Medicines 
Consortium's (SMC) decision to approve the use of NaMuscla(R) 
(mexiletine) for the treatment of symptomatic myotonia in adult patients 
with non-dystrophic myotonic disorders (NDM) in Scotland.(1) 
 
   NaMuscla is the first licensed medicine to treat symptomatic myotonia in 
adult patients with NDM(3) , an ultra-rare genetic neuromuscular 
disorder in which the sufferer's muscles are slow to relax after 
movement. These symptoms occur intermittently and unexpectedly, often 
causing pain, muscle weakness, fatigue and impairment of physical 
activities.(2) 
 
   "We are delighted that NaMuscla has been approved via the SMC fast track 
process and are pleased to offer a confidential patient access scheme to 
ensure patients in Scotland can access the only licensed medicine for 
this rare debilitating disease," said Ben Ellis, Lupin UK General 
Manager. "Scottish patients benefit from the SMC's tailored approach to 
rare disease medicine health technology assessments. Lupin is committed 
to ensuring all eligible patients can access NaMuscla, irrespective of 
where they live in the UK." 
 
   The SMC's approval applies only in the context of the agreed 
confidential NHS Scotland Patient Access Scheme (PAS). 
 
   For further information or media enquiries please contact: 
 
   Consilium Strategic Communications 
 
   Sukaina Virji / Lizzie Seeley 
 
   Tel: +44 (0)7738 499212 
 
   Email: lupin@consilium-comms.com 
https://www.globenewswire.com/Tracker?data=p7_GgryNyHTYfSMBSxhazr--2oBEETkyFYUjQ2H3jniNwG_ethx-TzlVH63ZRvwq9IWwCj_veu2HTWz-Js03z09r6v25gE6UkY835Th6fI6jpApSfrNviBTG1M6TassF 
 
 
   About Non-Dystrophic Myotonias (NDM) 
 
   Non-dystrophic myotonias (NDM) are a heterogenous group of rare 
neuromuscular disorders caused by mutations within ion channels in the 
sarcolemma membrane of skeletal muscles and affects 1 in 100,000 
people.(2) Non-dystrophic myotonias exhibit both sodium and chloride 
channelopathies which result in altered membrane excitability. The major 
clinical manifestation of the non-dystrophic myotonias is muscle 
stiffness as a consequence of the myotonia- delayed muscle relaxation 
after voluntary contraction. Additional common symptoms include pain, 
weakness and fatigue, and can affect different parts of the body, such 
as legs, arms or facial muscles, more severely.(2) 
 
   Myotonia in NDM patients has an onset in childhood and persists across 
their lifetime. Although not life limiting, patients with non-dystrophic 
myotonia can experience significant lifetime morbidity due to stiffness 
and pain related to myotonia. Patients may perceive that myotonia 
increases in severity over time, impacting 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.(7) 
 
   About mexiletine 
 
   In randomized controlled trials (3-5) mexiletine has been shown to 
significantly reduce myotonia compared to placebo in adult patients with 
NDM, reducing 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.The 
most commonly reported adverse reactions in patients treated with 
mexiletine are abdominal pain (12%), vertigo (8%) and insomnia (12%), 
demonstrating mexiletine has a good safety profile.(3,6) 
 
   About Lupin Limited 
 
   Lupin is an innovation-led transnational pharmaceutical company 
headquartered in Mumbai, India. The Company develops and commercializes 
a wide range of branded and generic formulations, biotechnology products 
and APIs in over 100 markets in the U.S., India, South Africa and across 
Asia Pacific (APAC), Latin America (LATAM), Europe and Middle East 
regions. 
 
   The Company supplies medicines in cardiovascular, anti-diabetic, and 
respiratory segments and has significant presence in the anti-infective, 
gastro-intestinal (GI), central nervous system (CNS) and women's health 
areas. Lupin is the 3rd largest pharmaceutical company in the U.S. by 
prescriptions and 5th in India by global revenues. The Company invests 
10.3 % of its revenues on research and development. 
 
   Lupin has fifteen manufacturing sites, seven research centers, more than 
20,000 professionals working globally.(8) 
 
   References 
 
 
   1. Scottish Medicines Consortium Final decision December 2020. 
      https://www.scottishmedicines.org.uk/medicines-advice/mexiletine-namuscla-resubmission-smc2307/ 
      https://www.scottishmedicines.org.uk/medicines-advice/mexiletine-namuscla-resubmission-smc2307/ 
      Last accessed 7/12/2020 
 
   2. Matthews E, Fialho D, Tan SV, Venance SL, Cannon SC, Sternberg D, et al. 
      The non-dystrophic myotonias: molecular pathogenesis, diagnosis and 
      treatment. Brain 2010; 133 (Pt 1): 9--22. 
 
   3. Namuscla summary of product characteristics, 
      https://www.medicines.org.uk/emc/product/9838/smpc#gref 
      https://www.medicines.org.uk/emc/product/9838/smpc#gref  Last accesses 
      Nov 2020 
 
   4. Statland JM, Bundy BN, Wang Y, et al. Consortium for Clinical 
      Investigation of Neurologic Channelopathies. Mexiletine for symptoms and 
      signs of myotonia in nondystrophic myotonia: a randomized controlled 
      trial. JAMA. 2012; 308(13):1357--1365. [PubMed: 23032552] 
 
   5. Stunnenberg B.C, Raaphorst J, Groenewoud H.M.et al. Effect of mexiletine 
      on muscle stiffness in patients with nondystrophic myotonia evaluated 
      using aggregated N-of-1 trials. JAMA. 2018; 320: 2344-2353 
 
   6. Suetterlin  KJ, Bugiardini  E, Kaski  JP,  et al.  Long-term safety and 
      efficacy of mexiletine for patients with skeletal muscle 
      channelopathies.  JAMA Neurol. 2015;72(12):1531-1533. 
 
   7. Trivedi JR, Bundy B, Statland J, et al; CINCH Consortium. Non-dystrophic 
      myotonia: prospective study of objective and patient reported outcomes. 
      Brain. 2013;136(pt 7):2189-2200. doi:10.1093/brain 
 
   8. https://wwww.lupin.com/wp-content/uploads/2020/05/lupin-q4fy20-investor-presentation.pdf 
      LUPIN Limited Investor Report FY2020 (Accessed Online: 3rdJuly 2020)

(END) Dow Jones Newswires

December 08, 2020 04:00 ET (09:00 GMT)