Adrenoleukodystrophy Drugs Market Report 2025

The adrenoleukodystrophy drugs market size has grown rapidly in recent years. It will grow from $0.63 billion in 2024 to $0.69 billion in 2025 at a compound annual growth rate (CAGR) of 10.1%. The growth in the historic period can be attributed to increased awareness of rare diseases, a rise in orphan drug designations, government funding for rare disease research, the emergence of personalized medicine, and expanding healthcare infrastructure.

The adrenoleukodystrophy drugs market size is expected to see rapid growth in the next few years. It will grow to $1.03 billion in 2029 at a compound annual growth rate (CAGR) of 10.4%. The growth in the forecast period can be attributed to increasing investment in rare disease research, rising adoption of precision medicine, growing healthcare expenditure in developing regions, improved newborn screening programs, and regulatory support for orphan drug development. Major trends in the forecast period include artificial intelligence (AI)-driven drug discovery, next-generation sequencing for early diagnosis, nanotechnology-based drug delivery, stem cell gene therapy innovations, and collaborations between biotech firms.

The forecast of 10.4% growth over the next five years reflects a modest reduction of 0.5% from the previous estimate for this market. This reduction is primarily due to the impact of tariffs between the US and other countries. Trade tensions could hinder U.S. rare neurology clinics by inflating prices of adrenoleukodystrophy therapies manufactured in Italy and Belgium, resulting in delayed neurodegenerative disease management and higher orphan drug costs. The effect will also be felt more widely due to reciprocal tariffs and the negative effect on the global economy and trade due to increased trade tensions and restrictions.

The growth of healthcare expenditure is a key driver of the adrenoleukodystrophy (ALD) drugs market. Healthcare expenditure, which includes the total financial resources allocated to medical services, healthcare products, and related services, is on the rise. This is largely due to an aging population and the growing prevalence of chronic diseases, both of which demand long-term care and continuous management. As a result, there is an increased need for specialized treatments for rare diseases, including ALD, which in turn leads to higher costs for research, development, and patient care. According to the Centers for Medicare and Medicaid Services (CMS), U.S. national health expenditures grew 7.5% in 2023, outpacing GDP growth, and are projected to grow at an annual rate of 5.6% from 2023 to 2032. As healthcare expenditure increases, so does the demand for treatments such as ALD drugs, fostering market growth.

Regulatory approvals from agencies such as the FDA and EMA are crucial for expanding the range of treatment options available for rare diseases such as ALD. These approvals ensure that treatments meet the necessary safety and efficacy standards, enabling their market introduction. A notable example is Minoryx Therapeutics, a Spain-based biotechnology company, which in May 2023 received FDA approval to initiate a Phase 3 clinical trial for leriglitazone (MIN-102) in adult male patients with X-linked adrenoleukodystrophy (X-ALD) and cerebral adrenoleukodystrophy (cALD). This clinical trial, known as CALYX, will assess the drug's ability to slow or halt disease progression, providing hope to patients with limited treatment options. A successful trial could lead to commercialization, addressing an urgent medical need for patients suffering from these devastating conditions.

In November 2022, Minoryx Therapeutics entered into a strategic partnership with Neuraxpharm Group to secure exclusive commercialization rights for leriglitazone in Europe. Neuraxpharm, a Germany-based pharmaceutical company specializing in treatments for central nervous system (CNS) disorders, including rare diseases such as X-ALD, will leverage its expertise in the European market to accelerate the commercialization of leriglitazone. This collaboration aims to improve patient access to this promising treatment and support the continued growth of the ALD drug market.

Major players in the adrenoleukodystrophy drugs market are Pfizer Inc., F. Hoffmann-La Roche AG, Sanofi S.A., AstraZeneca plc, Novartis AG, Teva Pharmaceuticals USA Inc., Dr. Reddy's Laboratories, Poxel S.A., Neuraxpharm, Mallinckrodt Pharmaceuticals plc, Nutricia, Viking Therapeutics Inc., MedDay Pharmaceuticals SAS, Voyager Therapeutics Inc., Prevail Therapeutics, Ashvattha Therapeutics, Magenta Therapeutics Inc., Minoryx Therapeutics S.L., SwanBio Therapeutics Inc., Autobahn Therapeutics Inc., bluebird bio Inc., SOM Biotech, and NeuroVia.

North America was the largest region in the adrenoleukodystrophy drugs market in 2024. The regions covered in adrenoleukodystrophy drugs report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the adrenoleukodystrophy drugs market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The adrenoleukodystrophy drugs market consists of sales of products including corticosteroids, Lorenzo's oil, hydrocortisone tablets, prednisone tablets, and adrenocortical hormone replacement therapy products. Values in this market are ‘factory gate’ values, that is, the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

Note that the outlook for this market is being affected by rapid changes in trade relations and tariffs globally. The report will be updated prior to delivery to reflect the latest status, including revised forecasts and quantified impact analysis. The report’s Recommendations and Conclusions sections will be updated to give strategies for entities dealing with the fast-moving international environment.

The sudden escalation of U.S. tariffs and the resulting trade tensions in spring 2025 are having a significant impact on the pharmaceutical sector. Companies are grappling with higher costs on imported active pharmaceutical ingredients (APIs), glass vials, and laboratory equipment - many of which have limited alternative sources. Generic drug manufacturers, already operating with minimal profit margins, are particularly affected, with some scaling back production of low-margin medications. Biotech firms are also experiencing delays in clinical trials due to shortages of specialized reagents linked to tariffs. In response, the industry is shifting API production to regions like India and Europe, building up inventory reserves, and advocating for tariff exemptions on essential medicines.

The adrenoleukodystrophy drugs market research report is one of a series of new reports that provides adrenoleukodystrophy drugs market statistics, including adrenoleukodystrophy drugs industry global market size, regional shares, competitors with an adrenoleukodystrophy drugs market share, detailed adrenoleukodystrophy drugs market segments, market trends and opportunities, and any further data you may need to thrive in the adrenoleukodystrophy drugs industry. This adrenoleukodystrophy drugs market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

Adrenoleukodystrophy (ALD) drugs are used to manage symptoms and slow the progression of ALD, a rare genetic disorder that impacts the nervous system and adrenal glands. This condition is caused by mutations in the ABCD1 gene, leading to the buildup of very long-chain fatty acids (VLCFAs) in the brain, adrenal glands, and spinal cord.

The primary drug categories for treating ALD include dietary supplements, antioxidants, corticosteroids, and enzyme replacement therapy. Dietary supplements are oral products containing essential nutrients such as vitamins, minerals, amino acids, and fatty acids, which support overall health and may help manage conditions such as ALD. These treatments are used for a range of conditions, including childhood cerebral adrenoleukodystrophy (CCALD), Addison's disease, and adrenomyeloneuropathy (AMN), and can be administered via oral, injectable, or intravenous routes. They are suitable for different age groups, including pediatric, adult, and geriatric populations, and are distributed through hospital pharmacies, retail pharmacies, online pharmacies, and other channels.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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