Albany NY, United States: Metachromatic leukodystrophy is a rare hereditary (genetic) disorder that causes accumulation of fatty substances (lipids) to build up in cells, particularly in the brain, spinal cord, and peripheral nerves.
Metachromatic Leukodystrophy Treatment: Clinical Trials Hold Significant Future Potential Treatment Options
- Albany NY, United States: Metachromatic leukodystrophy is a rare hereditary (genetic) disorder that causes accumulation of fatty substances (lipids) to build up in cells, particularly in the brain, spinal cord, and peripheral nerves. This buildup is caused by a deficiency of an enzyme called sulfatides that helps break down lipids.
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- The brain and nervous system progressively lose function because the substance that covers and protects the nerve cells (myelin) is damaged
- Metachromatic leukodystrophy is of three types: Late Infantile Form, Juvenile Form, and Adult Form
- This causes damage to the protective myelin covering the nerves, resulting in progressive worsening of brain and nervous system functions
- Treatment options include pipeline products which can cure only symptoms of disease. These products can produce effects such as prevention of nerve damage, slowing progression of the disorder, preventing complications, and providing supportive care.
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Key Drivers and Restraints of Global Metachromatic Leukodystrophy Treatment Market
- Increase in clinical trials with potential treatment options in coming years and increase in diagnosed cases are some of the major factors responsible for the growth of global metachromatic leukodystrophy market
- For instance, in 2019, Takeda Pharmaceutical Company Limited successfully initiated the phase II study of an Intrathecal SHP611 in participants with Late Infantile Metachromatic Leukodystrophy
- Rise in awareness about rare diseases in developed as well as developing regions is also expected to fuel the growth of global metachromatic leukodystrophy treatment market
- Moreover, development of novel drug molecules for the symptomatic relief of metachromatic leukodystrophy is expected to contribute toward the growth of global metachromatic leukodystrophy treatment market in the next few years
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Unavailability of Approved Therapeutic Options to Hamper Market Growth
- Not a single approved pharmacological product is available in the market for complete cure of metachromatic leukodystrophy which can hamper the market growth
- Higher cost of stem cell transplantation in some developing countries can also inversely impact the market growth
Pharmacotherapy to be Promising Treatment Type Segment
- Based on treatment type, the global metachromatic leukodystrophy treatment market can be segmented into pharmacotherapy and stem cell transplantation
- Stem cell transplantation accounted for major market share in 2019 as it is the only approved treatment option currently in the market
- Pharmacotherapy is expected to dominate the global market with higher CAGR during the forecast period due to several pipeline products which are expected to launch in coming years and novel mechanism of action for treatment
- Pharmacotherapy can be further divided into gene therapy, direct enzyme replacement, oligodendroglial cell therapy, and others
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Hospitals Dominated Sales Channel Segment
- In terms of sales channel, the global metachromatic leukodystrophy treatment market can be divided into hospitals, clinics, retail pharmacies, and others
- Hospitals segment dominated the global market in 2019 due to growing admission of patients for stem cell transplantation and availability of well-equipped staff
North America to Lead Global Metachromatic Leukodystrophy Treatment Market
- In terms of region, the global metachromatic leukodystrophy treatment market can be divided into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa
- North America dominated the global metachromatic leukodystrophy treatment market in 2019 due to higher number of diagnosed patients, ongoing clinical trials, and government funding for research & development for treatment options
- For instance, as per Orphanet, prevalence of Metachromatic Leukodystrophy in the U.S. is 1-9 per 1,000,000 people
- Asia Pacific is expected to be more lucrative segment during the forecast period, owing to rise in patient population, ongoing clinical trials, and growing awareness for rare diseases in the region
Key Manufacturers Operating in global Market
Key manufacturers operating in the global market include:
- Takeda Pharmaceutical Company Limited
- GlaxoSmithKline Plc
- ArmaGen Inc.
- RegenxBio Inc.
- Orchard Therapeutics plc
- Magenta Therapeutics
- Recursion Pharmaceuticals Inc.
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