Infantile Spasm Therapeutics Market – Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Therapeutic Class (Anticonvulsants, Corticosteroids, Others), By Drug Type (Vigabatrin, Adrenocorticotropic Hormone, Others (Phase III)), By Dosage (Solid, Liquid), by Region, and Competition, 2020-2030F

Market Overview

The Global Infantile Spasm Therapeutics Market , valued at USD 3.32 Billion in 2024, is projected to experience a CAGR of 4.04% to reach USD 4.21 Billion by 2030. Infantile spasm therapeutics comprise pharmaceutical agents specifically developed to treat infantile spasms, a severe and rare epileptic encephalopathy characterized by brief, abrupt muscle contractions often occurring in clusters in infants. Main market growth drivers include increasing global disease awareness, advancements in diagnostic methodologies enabling earlier detection, and the critical unmet medical need to prevent severe neurodevelopmental regression associated with the condition. According to StatPearls, updated in 2024, the incidence of infantile spasms ranges from 1.6 to 4.5 per 10,000 live births.

However, a significant challenge impeding market expansion is the high cost of existing first-line treatments, such as adrenocorticotropic hormone, coupled with often complex reimbursement processes. These factors can limit patient access to timely and effective therapies, especially in regions with developing healthcare infrastructures, thereby hindering overall market growth.

Key Market Drivers

The increasing prevalence of infantile spasms substantially drives the therapeutics market by expanding the treatable patient population. Improved diagnostic techniques and heightened awareness among medical professionals and caregivers facilitate earlier detection of these severe epileptic encephalopathies. Prompt diagnosis is critical for mitigating long-term neurodevelopmental consequences. According to data presented at the American Epilepsy Society in December 2024, from a cohort study, incidental diagnosis of Infantile Epileptic Spasms Syndrome (IESS) was observed in 29% of patients. This demonstrates how proactive screening brings more cases into the treatment pathway, directly fueling market demand.

Advancements in therapeutic interventions and drug development significantly propel the global infantile spasm therapeutics market. Ongoing research and development efforts introduce novel compounds and refine existing treatment modalities, offering improved efficacy, enhanced safety profiles, or more convenient administration. According to the American Epilepsy Society, in June 2024, the FDA approved Vigafyde, a new vigabatrin preparation, for infantile spasms as monotherapy in infants 1 month to 2 years of age. This innovation addresses unmet needs and refractory cases, spurring market expansion. Further, dedicated investment in the broader epilepsy landscape indirectly supports the infantile spasm sector. According to GlobeNewswire, in May 2023, Angelini Pharma announced an investment of USD 505.5 million and an upfront payment to JCR Pharmaceuticals Co. , Ltd. for novel epilepsy medicine development, signifying substantial financial commitment benefiting the specialized infantile spasms sector.

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Key Market Challenges

A significant challenge impeding the expansion of the Global Infantile Spasm Therapeutics Market stems from the high cost associated with existing first-line treatments, such as adrenocorticotropic hormone, coupled with often complex reimbursement processes. This financial burden critically limits patient access to timely and effective therapies, particularly in regions with developing healthcare infrastructures. The inability of patients to afford or readily obtain these essential medications directly translates into under-treatment or delayed intervention.

The substantial treatment costs directly hamper market growth by constraining demand and utilization of optimal therapeutics. For instance, according to a study published in *Epilepsia* in 2023, the use of ACTH as a first-line treatment for infantile spasms decreased from 78% in 2006 to 18% in 2020, reflecting a significant shift away from the higher-priced therapy as cost disparities with alternatives widened. Furthermore, according to the World Health Organization in August 2023, disparities in access to pediatric neurological care remain a key challenge in low-resource settings, where cost limitations often delay critical interventions for conditions like infantile spasms. This restricted access, driven by economic barriers and convoluted payment systems, ultimately hinders the overall growth potential of the market for infantile spasm therapeutics.

Key Market Trends

The Global Infantile Spasm Therapeutics Market is significantly shaped by the emergence of precision and genetically-targeted therapies. This trend represents a fundamental shift towards developing treatments that address the specific underlying genetic causes of infantile spasms, moving beyond broad-spectrum approaches. This individualized therapeutic strategy holds the promise of improved efficacy and reduced side effects for distinct patient populations. According to the American Epilepsy Society (AES), a study presented at their 2024 annual meeting highlighted that studies including non-European participants, representing only 16% of 68 genome-wide association studies, identified 58% of significant Single Nucleotide Polymorphisms linked to epilepsy. This demonstrates the impact of genetic research in uncovering crucial insights for targeted interventions. Furthermore, Neurocrine Biosciences was, as of November 2023, reviewing data for the potential implications of its NBI-921352 program for its ongoing study in SCN8A-developmental epileptic encephalopathy, a genetic form of epilepsy, showcasing active development in this specialized area.

Innovation in drug delivery and formulation formats is another pivotal trend influencing the infantile spasm therapeutics market. This involves developing medications in forms that are easier for infants to administer, improving patient adherence, and potentially enhancing therapeutic outcomes. Such advancements are critical given the challenges of administering medication to pediatric patients. According to the U. S. Food and Drug Administration (FDA), pediatric information was added to the labeling of 76 drugs and biologics in 2024, reflecting an increasing emphasis on tailoring medications for younger populations. This focus on child-friendly options is exemplified by Marinus Pharmaceuticals, which, as of October 2024, continued to support the commercial growth of ZTALMY® (ganaxolone) oral suspension CV, an FDA-approved medication for seizures associated with CDKL5 deficiency disorder in patients two years of age and older, with over 200 patients already receiving this specialized oral formulation.

Segmental Insights

The Anticonvulsants segment is demonstrating rapid growth in the Global Infantile Spasm Therapeutics Market, primarily driven by its established clinical efficacy in effectively reducing seizure frequency and severity. Medical practitioners favor these medications due to their generally favorable tolerability profiles and limited drug interactions, which collectively contribute to improved patient compliance. The versatility of anticonvulsants in addressing various seizure manifestations further positions them as a comprehensive therapeutic option. Moreover, continuous advancements in drug formulations enhance their effectiveness, while increasing global awareness and earlier diagnosis of infantile spasms stimulate the demand for prompt and effective treatment solutions.

Regional Insights

North America holds a leading position in the Global Infantile Spasm Therapeutics Market, driven by its well-established healthcare infrastructure and substantial investment in pharmaceutical research and development, which collectively foster innovation in rare disease therapies. The region benefits from a robust regulatory framework, including designations from the U. S. Food and Drug Administration (FDA) that expedite the approval of specialized treatments for conditions such as infantile spasms. Elevated awareness among healthcare professionals and parents, alongside widespread access to advanced diagnostic tools like EEG and neuroimaging, facilitates early detection and timely intervention. Additionally, the strong presence of specialized pediatric neurology networks and the availability of approved therapies further contribute to this regional dominance.

Recent Developments

• In October 2025, Ovid Therapeutics Inc. announced positive topline results from its Phase 1 healthy volunteer study for OV329, a next-generation GABA-aminotransferase inhibitor. This breakthrough research demonstrated strong inhibitory activity and a potentially improved safety profile compared to current treatments. OV329 was designed to avoid ocular accumulation, a known side effect of existing vigabatrin therapies for infantile spasms, as shown in previous preclinical studies where it did not accumulate in animal retinas. These findings support further development of OV329 for rare and drug-resistant forms of epilepsy, including infantile spasms.

• In August 2024, Mallinckrodt announced the availability of its Acthar Gel (repository corticotropin injection) Single-Dose Pre-filled SelfJect™ Injector in the U.S.. This new delivery device is designed to simplify the injection process for Acthar Gel, which is indicated for the monotherapy treatment of infantile spasms in infants and children under two years of age. The SelfJect injector aims to help ensure accurate dosing and includes enhanced safety features, such as a hidden needle. The U.S. FDA had approved Mallinckrodt's supplemental New Drug Application for SelfJect in February 2024.

• In June 2024, Pyros Pharmaceuticals received U.S. Food and Drug Administration (FDA) approval for Vigafyde, a new ready-to-use oral solution of vigabatrin, for the treatment of infantile spasms. This approval marked the first new drug formulation in this therapeutic area in fifteen years. Pyros Pharmaceuticals introduced Vigafyde, an oral solution specifically for pediatric patients aged one month to two years, with plans to launch it in the second half of 2024. The convenient, premeasured solution simplifies dosing for caregivers, aiming to reduce errors and improve treatment compliance for this severe seizure disorder. Vigafyde became available to patients in September 2024.

• In April 2024, Orphelia Pharma secured a European patent for KIGABEQ®, its innovative pediatric formulation of vigabatrin, an essential antiepileptic drug. The patent, entitled "Solid dosage forms of vigabatrin," and its granted U.S. counterpart, highlight the company's dedication to innovation in rare diseases. KIGABEQ® is available as 100 mg and 500 mg scored soluble tablets, specifically designed for oral or nasogastric administration in children aged one month to six years. This formulation is indicated for the first-line treatment of infantile spasms in Europe, offering a tailored option for this severe early childhood epilepsy.

Key Market Players

• Anavex Life Sciences Corp.
• H. Lundbeck A/S
• Catalyst Pharmaceuticals, Inc.
• Jazz Pharmaceuticals, Inc.
• Travere Therapeutics, Inc.
• Valerion Therapeutics LLC
• ORPHELIA Pharma
• Novartis AG
• Sanofi SA
• Pfizer Inc.

Report Scope:

In this report, the Global Infantile Spasm Therapeutics Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

• Infantile Spasm Therapeutics Market , By Therapeutic Class:

o   Anticonvulsants

o   Corticosteroids

o   Others

• Infantile Spasm Therapeutics Market , By Drug Type:

o   Vigabatrin

o   Adrenocorticotropic Hormone

o   Others (Phase III)

• Infantile Spasm Therapeutics Market , By Dosage:

o   Solid

o   Liquid

• Infantile Spasm Therapeutics Market , By Region:

o   North America

§  United States

§  Canada

§  Mexico

o   Asia Pacific

§  China

§  India

§  Japan

§  Australia

§  South Korea

o   Europe

§  France

§  United Kingdom

§  Italy

§  Germany

§  Spain

o   South America

§  Brazil

§  Argentina

§  Colombia

o   Middle East & Africa

§  South Africa

§  Saudi Arabia

§  UAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies presents in the Global Infantile Spasm Therapeutics Market .

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Company Information

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