Metachromatic leukodystrophy Treatment Market is Segmented By Type (Late-Infantile MLD, Juvenile MLD, Adult MLD), By Diagnosis (Blood tests, Genetic tests, Nerve conduction study, Magnetic resonance imaging (MRI), Psychological and cognitive tests, Others), By Treatment Type (Pharmacotherapy, Gene Therapy (AGT-183, GSK-2696274, Others), Enzyme replacement or enhancement therapy, Substrate reduction therapy, Others), By End-User (Hospital, Clinics, Diagnostic Centers, Others), and By Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) – Share, Size, Outlook, and Opportunity Analysis, 2023-2030
Metachromatic leukodystrophy treatment market is growing at a CAGR of 6.3% during the forecast period (2023-2030).
Metachromatic leukodystrophy is a rare hereditary (genetic) condition characterized by the accumulation of fatty substances (lipids) in cells, mainly in the brain, spinal cord, and peripheral nerves. A lack of an enzyme that helps break down lipids called sulfatides causes this accumulation. Because the substance that covers and protects nerve cells (myelin) is destroyed, the brain and nervous system gradually lose function. Metachromatic leukodystrophy is currently incurable. Early detection and treatment may help manage some signs and symptoms and postpone the progression of the illness, depending on the form and age of start.
As per DataM Intelligence, Metachromatic leukodystrophy Treatment Market study analysis offers an in-depth outlook on the market containing quantitative and qualitative data. It gives an outlook and forecast of the global market based on market segmentation. It also provides global Metachromatic leukodystrophy Treatment Market size, and growth, along with the latest trends, opportunities, and forecast till 2029 for the global market with esteem to major countries such as the United States, Canada, Brazil, Germany, Italy, Spain, United Kingdom, Russia, European countries, United Arab Emirates, Saudi Arabia, South Africa, Japan, China, India, South Korea, Australia, and rest of the countries over the globe.
Among all regions, the North American region is expected to hold the largest share of the global market over the forecast period. Metachromatic leukodystrophy Treatment Market in the United States and Canada produces the utmost share. Whereas the European Metachromatic leukodystrophy Treatment Market is projected to continue its presence globally during the period of 2022- 2029.
Metachromatic leukodystrophy Treatment Market Dynamics
Innovation in the products for treating demyelinating conditions is expected to drive market growth.
Joanne Kurtzberg an American professor of pediatrics and pathology at Duke University has developed a product called Duke O Cell or DUOC-01, which is a cell generated from umbilical cord blood that has been demonstrated to help people with demyelinating diseases. It's been used as a bridge therapy to help youngsters with inherited metabolic problems engraft faster. Moreover, Atidarsagene autotemcel (arsa-cel) is a gene therapy medicinal product based on autologous hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with a lentiviral vector encoding the ARSA cDNA driving supranormal ARSA expression in HSPCs and their progeny, with preliminary evidence of safety and efficacy in patients with early-onset MLD. Thus, from the above statements, the market is expected to drive in the forecast period.
Restraint:
The high cost of therapies for the treatment of Metachromatic leukodystrophy and there is no single FDA-approved pharmacological product on the market that can completely cure metachromatic leukodystrophy are the factors the market is expected to hamper in the forecast period.
COVID-19 Impact Analysis
The COVID-19 pandemic has moderately impacted healthcare systems and the market. Standard operating procedures for Personal Protective Equipment (PPE) for home care nurses were unclear during the pandemic. The doctors contacted all patients by email or telephone, as well as sent letters to those who did not have access to email or the internet. With little evidence of the impact of SARS-CoV-2 infections on rare diseases early in the pandemic, we decided that the danger of developing SARS-CoV-2 outweighed the risk of treatment interruption. Moreover, disruptions in therapy, whether planned or inevitable, resulted in a deterioration of clinical parameters and quality of life, as well as potentially negative outcomes such as cardiac events and/or renal function decline.
Additionally, the severity of the impact of the COVID-19 pandemic on business, including on preclinical and clinical development, its supply chain, and commercial programs got affected. Thus, from the above statements, the market got affected, and it is expected to gain traction quickly with the resumption of the economic activities.
Metachromatic leukodystrophy Treatment Market Segment Analysis
Gene Therapy segment is expected to hold the largest market share in metachromatic leukodystrophy treatment market
The gene therapy segment is expected to dominate in 2021. The segment's growth is beneficial because gene therapy showed benefits in pediatric patients with early-onset metachromatic leukodystrophy (MLD). There is currently no cure for MLD. In contrast, the European Medicines Agency (EMA) has recommended that the gene therapy Libmeldy, which consists of cryopreserved autologous CD34+ cells encoding the arylsulfatase-A gene, be given marketing authorization in the European Union to treat metachromatic leukodystrophy (MLD), a rare inherited metabolic disease that affects the nervous system. Libmeldy is for children who have been diagnosed as carriers of the faulty gene but have not yet shown symptoms and have the 'late infantile' or 'early juvenile' variants of MLD. It is used in children who have been diagnosed with the early juvenile form of the disease and have begun to show symptoms while still being able to walk independently and before cognitive impairment. Thus, from the above statements, the market segment is expected to hold the largest market share in the forecast period.
Metachromatic leukodystrophy Treatment Market
| Metrics | Details |
| Market CAGR | 6.3% |
| Segments Covered | By Type, By Diagnosis, By Treatment Type, By End-User, and By Region |
| Report Insights Covered | Competitive Landscape Analysis, Company Profile Analysis, Market Size, Share, Growth, Demand, Recent Developments, Mergers and acquisitions, New Product Launches, Growth Strategies, Revenue Analysis, and Other key insights. |
| Fastest Growing Region | Asia Pacific |
| Largest Market Share | North America |
Metachromatic leukodystrophy Treatment Market Geographical Analysis
North America region holds the largest market share in the global metachromatic leukodystrophy treatment market
In 2021, North America accounted for the highest revenue share. The increasing prevalence of metachromatic leukodystrophy, increased number of diagnosed patients, ongoing clinical trials, rise in government funding for research & development for the treatment, and launch of contact registry by the organizations in the region are some of the factors the market is expected to boost in the forecast period. For instance, metachromatic leukodystrophy ranges from 1/40,000 births in the United States of America. Moreover, The National Organization for Rare Disorders, Inc. has initiated a large-scale study to investigate Metachromatic Leukodystrophy (MLD), a brain disease that causes irreparable white matter destruction. The Metachromatic Leukodystrophy Community United Research and Engagement Survey (MLDCures) is a new project that provides a platform for Metachromatic Leukodystrophy patients worldwide to contribute information about the disease (MLD). Its goal is to create an international resource that scientists can use in future research. Additionally, Cure MLD is collaborating on the study with the National Organization for Rare Disorders (NORD), an independent organization that developed the IAMRARE platform to aid in the identification and treatment of all 7,000 rare diseases. Thus, from the above statements, the North American region is expected to hold the largest market share in the forecast period.
Competitive Landscape
Major key players in the metachromatic leukodystrophy treatment market are ArmaGen, Inc., GlaxoSmithKline plc, Takeda Pharmaceutical Company Limited, Orchard Therapeutics plc, Magenta Therapeutics, Recursion Pharmaceuticals Inc. and RegenxBio Inc.
ArmaGen, Inc.:
Overview:
ArmaGen is a privately held, clinical-stage biotechnology company, with headquarters and research operations located in Calabasas, CA focused on developing revolutionary therapies for severe neurological disorders. The company is advancing innovative therapies for the treatment of currently unaddressed neurological complications of lysosomal storage disorders (LSDs), as well as neurodegenerative diseases such as Alzheimer’s and Parkinson’s. Moreover, ArmaGen has eight compounds in development. The company’s lead product candidates are AGT-182 for Hunter syndrome, which is in Phase 1 clinical trials and AGT-181 for Hurler syndrome, which is in Phase 2 trials. The company is advancing its pipeline through licensing and collaboration agreements, in-house development efforts, and future partnering opportunities.
ÂProduct Portfolio:
AGT-183: AGT‑183 is an investigational enzyme replacement therapy (ERT) for the treatment of metachromatic leukodystrophy (MLD), a lysosomal storage disease that arises from a deficiency of the enzyme arylsulfatase (ASA).
