United States Rare Neurological Disease Drugs Market 2025 | Growth Drivers, Key Players & Investment Opportunities

Market Size and Growth

The Global Rare Neurological Disease Drugs Market reached US$ 139.4 billion in 2023 and is expected to reach US$ 269.7 billion by 2031 growing with a CAGR of 8.6% during the forecast period 2024-2031.Key Development:

Key Development:

United States: Recent Industry Developments

✅ In September 2025, uniQure's gene therapy AMT-130 demonstrated a 75% reduction in disease progression over three years in Huntington's disease patients, with plans for FDA submission in early 2026.

✅ In September 2025, Ionis Pharmaceuticals announced positive Phase 3 results for zilganersen, an investigational drug showing improved mobility in patients with Alexander disease, a rare neurological condition.

✅ In September 2025, the FDA granted accelerated approval to Stealth BioTherapeutics' FORZINITY (elamipretide), marking the first treatment for Barth syndrome, a rare mitochondrial disorder.

Japan: Recent Industry Developments

✅ In September 2025, Chugai Pharmaceutical filed for an additional indication of Avastin for the treatment of Neurofibromatosis Type 2, aiming to address a rare intractable disease with limited treatment options.

✅ In June 2025, Sanofi's riliprubart received orphan drug designation from Japan's Ministry of Health for treating Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), a rare neurological disorder.

✅ In June 2025, Takeda launched LIVMARLI® Oral Solution for treating pruritus associated with cholestasis in Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis, becoming the first approved therapy for these conditions in Japan.

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Rare Neurological Disease Drugs are therapeutic medications developed to treat uncommon neurological disorders, such as Huntington's disease, spinal muscular atrophy, or certain neuropathies. These drugs aim to manage symptoms, slow disease progression, and improve quality of life, often leveraging advanced approaches like gene therapy, biologics, or orphan drug frameworks.

Recent Mergers and Acquisitions:

United States: Recent M&A Developments

✅ In May 2025, Johnson & Johnson announced the acquisition of Intra-Cellular Therapies for $14.6 billion, enhancing its neuroscience portfolio with Caplyta, a treatment for schizophrenia and bipolar depression.

✅ In April 2025, Merck KGaA finalized a $3.9 billion acquisition of SpringWorks Therapeutics, bolstering its rare cancer therapies portfolio, particularly in treating desmoid tumors.

✅ In April 2025, Novartis agreed to acquire Regulus Therapeutics for up to $1.7 billion, aiming to strengthen its RNA-targeted therapies pipeline, including treatments for rare kidney diseases.

Japan: Recent M&A Developments

✅ In June 2025, Sanofi acquired Blueprint Medicines for $9.1 billion, expanding its portfolio in rare immunological diseases and adding Ayvakyt, the first approved treatment for systemic mastocytosis.

✅ In May 2025, Sanofi completed the acquisition of Vigil Neuroscience for $470 million, adding Alzheimer's candidate VG-3927 to its neurology pipeline.

Key Players:

Novartis AG, Pfizer, Inc., Johnson & Johnson Services, Inc., US WorldMeds LLC (Solstice Neurosciences LLC), Aquestive Therapeutics Inc., Sanofi S.A., Merck & Co., Inc., CSL Ltd, Merz Pharma GmbH & Co. KGaA, and Kedrion Biopharma Inc. among others.

Growth Forecast Projected:

The Global Rare Neurological Disease Drugs Market is anticipated to rise at a considerable rate during the forecast period, between 2024 and 2031. In 2023, the market is growing at a steady rate, and with the rising adoption of strategies by key players, the market is expected to rise over the projected horizon.

Research Process:

Both primary and secondary data sources have been used in the global Rare Neurological Disease Drugs Market research report. During the research process, a wide range of industry-affecting factors are examined, including governmental regulations, market conditions, competitive levels, historical data, market situation, technological advancements, upcoming developments, in related businesses, as well as market volatility, prospects, potential barriers, and challenges.

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Key Segments:

➥ By Drug Type: Biologics, Small Molecules

➥ By Route of Administration: Intravenous, Oral

➥ By Application: Dravet Syndrome, Adrenoleukodystrophy, Narcolepsy, Angelman Syndrome, Amyotrophic Lateral Sclerosis, Others

➥ By Distribution Channel: Hospital Pharmacies, Retail Pharmacies, Online Pharmacies

Regional Analysis for Rare Neurological Disease Drugs Market:

⇥ North America (U.S., Canada, Mexico)

⇥ Europe (U.K., Italy, Germany, Russia, France, Spain, The Netherlands and Rest of Europe)

⇥ Asia-Pacific (India, Japan, China, South Korea, Australia, Indonesia Rest of Asia Pacific)

⇥ South America (Colombia, Brazil, Argentina, Rest of South America)

⇥ Middle East & Africa (Saudi Arabia, U.A.E., South Africa, Rest of Middle East & Africa)

Benefits of the Report:

Chapter 1: Sets the stage by outlining the report's coverage, summarizing key market segments by region, product type, and application. Presents a snapshot of market sizes, growth potential across segments, and anticipated industry evolution both short and long term.

Chapter 2: Highlights pivotal market insights and uncovers the most significant emerging trends driving change within the industry.

Chapter 3: Offers an in-depth look at the competitive landscape among Rare Neurological Disease Drugs producers, including revenue shares, strategic moves, and recent mergers and acquisitions.

Chapter 4: Presents comprehensive profiles of the market's key players, delving into details such as revenue, profit margins, product portfolios, and company milestones.

Chapters 5 & 6: Analyze Rare Neurological Disease Drugs revenue at both regional and country levels, providing quantitative breakdowns of market sizes, growth opportunities, and development prospects worldwide.

Chapter 7: Focuses on different market segments by type, examining their individual sizes and potential, guiding readers toward high-impact, untapped market areas.

Chapter 8: Explores segmentation by application, evaluating industry growth potential in various downstream markets and pinpointing promising sectors for expansion.

Chapter 9: Provides a thorough review of the industry's supply chain mapping out both upstream and downstream activities.

Chapter 10: Concludes with a summary of the report's key findings and highlights the most critical takeaways for industry stakeholders.

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FAQ

What is the current size of the Rare Neurological Disease Drugs Market?

A: In 2023, the Rare Neurological Disease Drugs Market was valued at US$ 139.4 billion, reflecting its strong industry presence.

Q2: How large is the Rare Neurological Disease Drugs Market expected to be by 2031?

A: By 2031, industry forecasts suggest the Rare Neurological Disease Drugs Market will grow to around US$ 269.7 billion, demonstrating significant expansion.

Q3: What is the growth rate of the Rare Neurological Disease Drugs Market?

A: The market is projected to expand at a compound annual growth rate (CAGR) of 8.6% during the forecast period from 2024 to 2031.

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