Report Overview

According to the deep-dive market assessment study by Growth Plus Reports, the global neurometabolic disorders market is expected to register a revenue CAGR of 5.2% during the forecast period from 2022 to 2030. 

Neurometabolic Disorders Market Fundamentals

Neurometabolic diseases are a group of genetically defined diseases that affect all organs and are identified using biochemical tests performed on a few accessible tissues. Neurometabolic diseases are triggered by disruptions in neurotransmitter metabolism, which include amino acids, such as aminobutyric acid (GABA), glycine, and glutamate, and cholinergic transmission, and monoamines, such as dopamine, serotonin, noradrenaline, adrenaline, and purines. The pathophysiology of neurological illnesses is also influenced by other molecules, such as neuropeptides, ion channels, and numerous neuromodulators engaged in neurotransmission pathways.

Neurometabolic Disorders Market Dynamics 

Increasing prevalence of various neurometabolic disorders globally is the major factor driving the revenue growth of the market. Moreover, rise in the demand for early detection and treatment of neurometabolic diseases is also expected to boost revenue growth of the neurometabolic disorders market. Furthermore, increase in initiatives by several government and non-government organizations to raise awareness regarding this disorder is also contributing to market revenue growth.

Rising R&D activities focused on the development of effectual treatment solutions for neurometabolic disorders, incorporation of next-generation sequencing (NGS), and rising technological advancements in gene therapy and metabolic treatment, together with a robust product pipeline, are expected to boost the market revenue growth in the near future.

However, lack of awareness in developing countries, existence of a limited patient population to determine the best dosage for treating the disorder, and high cost associated with research are expected to restrict the market revenue growth to some extent.

Neurometabolic Disorders Market Ecosystem

The global neurometabolic disorders market is analyzed from three perspectives: type, route of administration, and region.

Neurometabolic Disorders Market by Type

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Based on the type, the global neurometabolic disorders market is segmented into Gaucher's disease (GD), Fabry disease, Pompe disease, mucopolysaccharidosis VI, Niemann-pick type c disease, and others. 

The Gaucher's disease segment accounts for the dominant revenue share of the neurometabolic disorders market. This can be attributed to rise in the prevalence of GD. According to the National Center of Biotechnology Information, the cases of GD ranged from 0.70 to 1.75 per 100,000 globally. Additionally, increase in cases of different diseases associated with GD, such as cardiovascular diseases and CNS-related disorders, further drive revenue growth in the segment. At present, enzyme replacement therapies (ERT) and substrate reduction therapies (SRT) are two treatment solutions available that are approved for Gaucher's disease. Thus, major players are investing heavily in the development of new drugs for the treatment of GD. This factor is expected to fuel revenue growth in the segment during the forecast period. 

Neurometabolic Disorders Market by Route of Administration

Based on the route of administration, the global neurometabolic disorders market is segmented into oral and parenteral. 

The parenteral segment accounts for the largest revenue share of the market as the majority of treatments for neurometabolic disorders are enzyme- or gene-based, and parenteral is the most preferred route of administration in neurometabolic disorders. Moreover, this route of administration also enables precise drug targeting and prolonged drug release, which is also a key factor supporting the large revenue share of the segment.

Neurometabolic Disorders Market by Region

Based on the region, the global neurometabolic disorders market is segmented into North America, Europe, Asia Pacific, and the rest of the world. 

North America dominates the global neurometabolic disorders market, followed by Europe. The Asia Pacific market is expected to register the fastest revenue CAGR during the forecast period, mainly due to a well-developed biopharmaceutical industry in the region. Moreover, rising R&D activities in genomic-based biopharmaceutical and several other research institutes are playing a significant role in the regional market revenue growth. Additionally, support provided by the FDA to the companies in this region is also expected to support the market expansion in this region. The benefits include market exclusivity for diagnostics and pharmaceutical companies engaged in the production of orphan drugs, tax credits for approved clinical testing, and exemption from prescription drug payments for orphan drugs.

Neurometabolic Disorders Market Competitive Landscape

The prominent players operating in the global neurometabolic disorders market are:

• Amicus Therapeutics
• ISU Abxis
• JCR Pharmaceuticals
• Biosidus SA
• Greenovation Biotech
• UAB Proforma
• Dong-A Socio Group
• ExSAR Corporation
• Neuraltus Pharmaceuticals
• Greenovation Biotech
• Shire Plc (Takeda Pharmaceutical Company)
• Lixte Biotechnology Holdings, Inc.
• Pharming Group N.V.
• Protalix BioTherapeutics
• Biomarin Pharmaceutical Inc.

Neurometabolic Disorders Market Strategic Developments

• In May 2022, Liberyx Therapeutics announced the completion of its series A fundraising to support the further development of multiple pipeline programs ready for clinical development. The company has multiple promising candidates for rare neurometabolic disorders.
• In April 2022, POXEL SA received FDA Fast Track Designation (FTD) to PXL770 for the treatment of patients with adrenomyeloneuropathy (AMN), the most common form of X-linked adrenoleukodystrophy (ALD), which is an orphan neurometabolic disease caused by mutations in the ABCD1 gene. PXL770 is a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator.
• In October 2021, Takeda Pharmaceutical Company collaborated with Immusoft to discover, develop, and commercialize transformative cell therapies in rare inherited metabolic disorders with the central nervous system (CNS) and innovative therapeutics for rare neurometabolic diseases using Immusoft’s Immune System Programming (ISP). This technology platform modifies a patient’s B cells and instructs the cells to deliver gene-encoded therapies.

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