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

Tue 16th February, 2021 7:00am
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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

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.”

Lupin has provided a comprehensive data package to demonstrate the long term
safety and efficacy of NaMuscla, and the significantly improved quality of
life(4) for NDM patients. Supporting evidence includes three randomised
controlled studies(4-6) and two long term studies.(7,8) Lupin continues to
invest in this disease area and has further investment commitments for
NaMuscla including, but not limited to, a long-term safety study for adults
patients and a trial for paediatric patients.

For further information or media enquiries please contact:

Consilium Strategic Communications 
Sukaina Virji / Lizzie Seeley /Kris Lam
Tel: +44 (0)20 3709 5700

Notes for Editors

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( 9) . 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.(10)

About mexiletine
In randomized controlled trials(, )(4-6)  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%) (4)., demonstrating mexiletine has a
good safety profile .

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 in FY2020 on research and development.
Lupin has fifteen manufacturing sites, seven research centers, more than
20,000 professionals working globally(11)
1. NICE Appraisal consultation document Mexiletine for treating myotonia in
adults with non-dystrophic myotonic disorders Feb 2021
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. Orphan maintenance assessment report, Namuscla:
Last accessed Nov 2020
4. Namuscla summary of product characteristics,  Last accessed Nov
5.  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]
6. Stunnenberg B.C, Raaphorst J, Groenewoud al. Effect of mexiletine on
muscle stiffness in patients with nondystrophic myotonia evaluated using
aggregated N-of-1 trials. JAMA. 2018; 320: 2344-2353
7. 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.
8. Lupin Limited. Long term follow up data from MYOMEX - EMA response Q13
Annex. LUP-NAM-065. Data on file. 2019
 1. Trip J, Drost G, Ginjaar HB, Nieman FH, van der Kooi AJ, de Visser M, et
al. Redefining the clinical phenotypes of non-dystrophic myotonic
syndromes. J Neurol Neurosurg Psychiatry. 2009;80:647–52. [PubMed
(] [Google Scholar
 1. Trivedi JR, Bundy B, Statland J, et al; CINCH Consortium.
Non-dystrophicmyotonia: prospective study of objective and patient reported
outcomes. Brain. 2013;136(pt 7):2189-2200.
LUPIN Limited Investor Report FY2020 (Accessed Online: 3rdJuly 2020)
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