Enables expanded access to the only EU-approved treatment for myotonia symptoms
in adults with non-dystrophic myotonic disorders

Mumbai, Zug, January 28, 2025: Global pharma major Lupin Limited (Lupin) and Avas Pharmaceuticals SRL (Avas) today announced the launch of Lupin’s orphan drug NaMuscla® (mexiletine) in Italy. Avas will commercialize NaMuscla® for the symptomatic treatment of myotonia in adults with non-dystrophic myotonic (NDM) disorders. NaMuscla® is the first and only licensed product for this indication.

NDM disorders are a group of rare, inherited neuromuscular disorders which is characterised by the inability to relax muscles following voluntary contraction. NaMuscla® reduces myotonia symptoms in people with NDM, resulting in a significant improvement in quality of life and other functional and clinical outcomes for patients¹. NaMuscla®, which has been designated orphan drug status, received EU marketing authorization in December 2018 ².

Under the terms of the agreement, Avas will initially commercialize NaMuscla® as a Class C (Tier 2), while Lupin negotiates with AIFA (Agenzia Italiana del Farmaco) for the reimbursement of the product in Italy. This will ensure consistent access to NaMuscla® for patients in Italy, leveraging the established supply network that already serves patients across Germany, France, Spain, Austria, Norway, Sweden, and the UK.

“Our collaboration with Avas will not only allow us to introduce the first licensed treatment for myotonia in Italy but also ensure continued access to this essential medication. This partnership reflects Lupin’s dedication to addressing unmet medical needs in rare diseases across Europe,” stated Thierry Volle, President EMEA, Lupin.

“We are excited to announce our partnership with Lupin for the launch of NaMuscla^® in Italy, a key step in addressing the needs of patients with NDM disorders. This collaboration ensures the availability of best therapeutic solutions, improving the quality of life for individuals affected by NDM disorders in Italy,” said Marco Rago, President of the Board of Directors at Avas Pharmaceuticals.

Notes for Editors

About Myotonic Disorders and Non-Dystrophic Myotonias (NDM)
Myotonic disorders are a group of heterogeneous, inherited, neuromuscular disorders characterized by a shared symptom called myotonia³. Myotonia can be described as an inability to relax a contraction of skeletal muscle which originates from a voluntary muscular contraction such as shaking someone’s hand and blinking, or everyday activities such as walking across a street and climbing stairs⁴.

Non-dystrophic myotonias (NDM) are a sub-set of rare (prevalence of 1:100,000⁴), 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 excitability⁵. For patients with NDM, myotonia is the most prominent symptom and demonstrates different phenotypes in subgroups of NDM disorders, and can affect different parts of the body, such as legs, arms, or facial muscles, more severely⁵.

Myotonia in patients with NDM has an onset in childhood and persists across their lifetime. Patients 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^{3, 6}.

About NaMuscla® (mexiletine)
NaMuscla® is the first and only antimyotonic agent licensed to treat symptomatic myotonia in adults with non-dystrophic myotonic disorders in Europe². In randomised controlled trials^{1, 3, 6}, NaMuscla® (167 to 500 mg/day) has been shown to significantly reduce myotonia compared to placebo, 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, with gastro-intestinal discomfort reported as the most common adverse event, demonstrating NaMuscla® to be safe and well tolerated^{1, 3, 6,7}.

About Lupin
Lupin Limited is a global pharmaceutical leader headquartered in Mumbai, India, with products distributed in over 100 markets. Lupin specializes in pharmaceutical products, including branded and generic formulations, complex generics, biotechnology products, and active pharmaceutical ingredients. Trusted by healthcare professionals and consumers globally, the company enjoys a strong position in India and the U.S. across multiple therapy areas, including respiratory, cardiovascular, anti-diabetic, anti-infective, gastrointestinal, central nervous system, and women’s health. Lupin has 15 state-of-the-art manufacturing sites and 7 research centers globally, along with a dedicated workforce of over 22,000 professionals. Lupin is committed to improving patient health outcomes through its subsidiaries – Lupin Diagnostics, Lupin Digital Health, and Lupin Manufacturing Solutions.

To know more, visit www.lupin.com or follow us on LinkedIn https://www.linkedin.com/company/lupin

For further information or queries please contact:

Rajalakshmi Azariah
Vice President & Global Head – Corporate Communications, Lupin
rajalakshmiazariah@lupin.com

About Avas
AVAS Pharmaceuticals is a leading pharmaceutical company renowned for its expertise in the commercialization of branded medicines that offer transformative therapeutic solutions. With a deep commitment to improving patient outcomes, AVAS Pharmaceuticals is dedicated to providing innovative treatments that have the potential to significantly impact patients’ lives across Italy.

Founded on principles of innovation, excellence, and accessibility, AVAS Pharmaceuticals operates in a diverse range of therapeutic areas, including oncology, cardiovascular health, infectious diseases, and neurology. The company also specializes in addressing rare and ultra-rare diseases, offering life-changing medicines for patients with limited or no therapeutic options.

AVAS Pharmaceuticals’ dedication to quality, innovation, and patient care positions it as a key player in the Italian healthcare landscape, and its continued growth is a testament to the transformative impact it has on patients and their families.

Contact for media inquiries:

administration@avaspharma.com

References:

1. Vicart S, et al. Neuromuscular Discord 2021; 31:1124-1135
2. NaMuscla®. Summary of Product Characteristics. https://www.ema.europa.eu/en/medicines/human/EPAR/namuscla#product-info
3. Stunnenberg, et al. Muscle Nerve 2020;62(4):430-444
4. Emery AEH, Neuromuscular Discord 1991;1:19-29
5. Matthews E, et al. Brain 2010;133:9–22
6. Trivedi JR, et al. Brain 2013; 136:2189-200
7. Matthews E, et al. Pract Neurol 2021;0:1–10