Servier – Our commitment to neurology

neurology

A commitment to addressing major medical needs in rare diseases

Rare neurological diseases affect between 130 and 220 million people¹, with around 600 rare neurological disorders identified². Despite their “rare” status individually, their collective burden is massive. At Servier, we focus on creating new medicines that slow down or halt the progression of rare neurological disorders and significantly improve patient quality of life.

A societal imperative and scientific challenge

Demographic trends, advances in lifespan and genotyping are driving these numbers upward with a prevalence that could increase up to 50% between now and 2040 leading to up to 400 million people concerned ³.

These diseases are frequently underdiagnosed, poorly understood, and lack effective treatments. Critically, it is estimated that ~90% of rare neurological diseases currently have no disease-modifying therapy available ⁴.

Did you know?

The economic and social burden is 5 to 7 times higher per patient compared to common chronic diseases⁵. This encompasses direct medical costs, loss of productivity, and the profound emotional impact on families and caregivers.

Therefore, addressing rare neurological diseases is not only a scientific and medical challenge, but also a societal imperative. Advancing research and innovation in this field holds the promise of transforming lives, driving medical breakthroughs, and ensuring that no patient is left behind simply because their disease is rare.

What are our focus areas?

Despite the growing medical need, only a limited number of treatments are available to alter the progression of these disorders or alleviate symptoms significantly. We are committed to addressing this major unmet medical need in neurology with a particular focus on clusters within rare neurological diseases. In addition, we aligned these disease areas with our scientific strategy, leveraging the expertise and technological platforms we can deploy effectively (small molecules, monoclonal antibodies and antisense oligonucleotides). We therefore defined six clusters of diseases:

Rare refractory epilepsies refer to a group of uncommon epilepsies syndromes that are resistant to standard anti-seizure medications and often have a genetic or structural origin. These conditions typically begin in infancy or childhood, are associated with severe seizures, and can lead to neurodevelopmental regression, cognitive impairment, and significant morbidity. Some forms are progressive and life threatening⁶.
Rare movement disorders are an expansive and diverse group of uncommon neurological conditions, often genetic or inherited, that typically begin in childhood or early adulthood. They are marked by abnormal involuntary movements such as tremors, dystonia, chorea, ataxia or paroxysmal dyskinesias, and are frequently progressive and disabling with limited treatment options⁷. Examples: Friedreich’s ataxia and spinocerebellar ataxia.
Neuromuscular disorders areoften genetic or immune-mediated conditions that impair the motor neurons, peripheral nerves, neuromuscular junctions, or muscle fibers. They present progressive muscle weakness, muscle wasting, and functional impairment. They are chronic and debilitating, and for many neuromuscular disorders, no curative treatment is available⁸. Examples: amyotrophic lateral sclerosis and autoimmune myasthenia gravis.
Genetically-driven autism spectrum disorders (ASD) refer to a form of ASD characterized by a clear genetic etiology, typically monogenic or syndromic, often involving single gene mutation or copy-number variants (CNVs). These syndromes present early in development and are frequently accompanied by intellectual disabilities, occasional epilepsy, and other neurological features. They represent a distinct group within ASD, where genetic diagnosis informs prognosis, counseling and emerging precision medicine therapies⁹. Examples: Phelan McDermid Syndrome, Fragile X Syndrome.
Leukodystrophies are a group of rare, inherited disorders characterized by the abnormal formation or the progressive degeneration of the white matter (myelin sheet) in the brain and spinal cord. They typically begin in childhood and lead to progressive neurological decline and are often fatal due to a lack of curative treatments¹⁰. Examples: adrenoleukodystrophy, Nieman-Pick Disease.
Peripheral neuropathies are a heterogenous group of uncommon, often inherited, or immune-mediated disorders that affect the peripheral nervous system. These diseases typically involve progressive damage to motor, sensory or autonomic nerves, leading to symptoms such as muscle weakness, sensory loss, neuropathic pain, and mobility impairment. Many are chronic and debilitating, and currently incurable. Some can be life-threatening. Examples: Charcot-Marie-Tooth disease, Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP).

What do all these disorders have in common?

They are generally early-onset, genetically driven conditions that lead to neurodevelopmental, cognitive or progressive neurological impairment. These diseases are all severe and debilitating; some are fatal, especially in pediatric forms with rapid progression. Despite their low individual prevalence, they collectively represent a significant burden for patients and families due to their chronic, disabling nature and the lack of curative therapies.

Managing these disorders requires highly specialized, multidisciplinary care. Scientifically, they present strong opportunities for innovation through precision medicine, including mRNA therapies, biomarkers-driven approaches, and digital health technologies.

Going the extra mile toward new treatments in neurology

In R&D, we focus on creating new medicines that slow down or halt the progression of rare neurological disorders and significantly improve patient quality of life.

Learn more about our ambition in R&D

Our scientific focus targets a limited number of mechanisms of action common to these pathologies, enabling us to develop meaningful strategies to fight their progression. Our approach is grounded in data from patients, together with a strong degree of biological, immunological and molecular evidence, which provides a robust scientific rationale for addressing these disorders.

How we fight neurological disorders

Our neurology research is focused on:

Antisense oligonucleotides – Short synthetic modified sequences of DNA or RNA, called nucleotides, selectively target RNA to alter their protein expression. To find out more about our in-house efforts to improve and leverage this technology in the area of neurology, read our insight. Read the insight on ASO.
Small molecules – Servier leverages its historical experience in small molecule design to develop innovative therapies that inhibit the activity of pathological proteins that cause disease. More recently, Servier has expanded its scope to small molecules targeting RNA to modulate protein expression.
Monoclonal antibodies – Therapeutic antibodies are specifically selected and designed to bind to and modify the activity of disease-related targets. Monoclonal antibodies represent one of the most important global pharmacological advances of the past two decades and Servier-Symphogen is a serious player in the design of monoclonal antibodies whether they are mono, bi or tri specific. What are they exactly, and what roles do they play? Get some answers in our insight. Read the insight about mAb.

In June 2025, our neurology pipeline comprised 9 projects, 2 of which in clinical development and 7 research projects.
Learn more about our pipeline

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[1] Mancuso et al. Neurol Sci 2020 ; Frontiers in Neurology April 2023 – https://pubmed.ncbi.nlm.nih.gov/31989346/
[2] Rare neurological disorders markets, DelveInsight Business Research, Jan 2025 – https://www.globenewswire.com/news-release/2025/01/09/3007280/0/en/Latest-Published-4-Rare-Neurological-Disorders-Market-Reports-by-DelveInsight-Amyotrophic-Lateral-Sclerosis-Developmental-and-Epileptic-Encephalopathies-Primary-Ciliary-Dyskinesia-.html
[3 ] Global Burden of Disease 2021 Nervous systems Disorders Collaborators, The Lancet Neurology, 23(4); J. Lei & K Gillepsie et al., Neurology, April 2024 – https://pmc.ncbi.nlm.nih.gov/articles/PMC10949203/
[4] NORD, Rare Disease Facts and Figures, 2024 – https://rarediseases.org/understanding-rare-disease/rare-disease-facts-and-statistics/
[5] Orphanet Journal of Rare Diseases, 2023
[6] Chin et al., Frontiers in Neurology, 2021; Goodspeed et al., Frontiers in Neurology, 2022 – https://www.frontiersin.org/journals/neurology/articles/10.3389/fneur.2022.805007/full
[7] www.movementdisorders.org
[8] https://my.clevelandclinic.org/health/diseases/neuromuscular-disorders
[9] Leblond et al. Annual review of genetics vol 58, 2024 – https://www.annualreviews.org/content/journals/10.1146/annurev-genet-111523-102614; Halaweh, clin Neurol and neurosc. 8(4) 2024 – https://www.sciencepublishinggroup.com/article/10.11648/j.cnn.20240804.11
[10] McCoy et al., EBSCO website, 2024

Neurology