Adrenoleukodystrophy Market
Number of pages: 100 | Report Format: PDF | Published date: March 03, 2023
Historical Years – 2021 | Base Year – 2022 | Forecasted Years – 2023-2031
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Report Attribute |
Details |
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Market Size Value in 2022 |
US$ 935.21 million |
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Revenue forecast in 2031 |
US$ 1,945.51 million |
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CAGR |
7.6% |
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Base Year for Estimation |
2022 |
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Forecast Period |
2023-2031 |
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Historical Year |
2021 |
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Segments Covered |
Diseases Type, Treatment, End-user, and Region |
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Regional Scope |
North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa |
According to the deep-dive market assessment study by Growth Plus Reports, the global adrenoleukodystrophy market was valued at US$ 935.21 million in 2022 and is expected to register a revenue CAGR of 7.6% to reach US$ 1,945.51 million by 2031.
Adrenoleukodystrophy Market Fundamentals
Adrenoleukodystrophy, or ALD, is an X chromosome-linked hereditary disease. It affects both the nervous system and the adrenal organs. Saturated, very long-chain fatty acids accumulate in the brain, nerve system, and adrenal gland in people with adrenoleukodystrophy, as the bodies cannot break down these long-chain fatty acids. The fatty membrane (myelin) that protects the brain and spinal cord nerves tends to degrade in this disease (demyelination). The capacity of the nerves to transmit information to the brain is weakened when myelin is depleted. Additionally, a lack of certain hormones is brought on by damage to the adrenal cortex, the outer covering of the adrenal glands (adrenocortical insufficiency).
Weakness, weight loss, facial changes, vomiting, and coma may be indications of adrenocortical insufficiency. Changes in cognition and conduct are common symptoms of adrenoleukodystrophy. Men are more prone to adrenoleukodystrophy than women. Treatment aims at reducing or preventing the disease’s symptoms and progression. Adrenoleukodystrophy has no known treatment. With the recent implementation of newborn screening (NBS) for adrenoleukodystrophy, the disease can be identified before it manifests clinically, providing an opportunity for further phenotyping and genetic characterization. A promising new therapy for childhood cerebral adrenoleukodystrophy (ccALD) is developing that combines gene therapy and autologous hematopoietic stem cell transplantation. The clinical management and outcomes of adrenoleukodystrophy are changing due to these advancements in adrenoleukodystrophy screening and therapy.
More research is needed to determine the best treatment approach for women with adrenoleukodystrophy, including timing and clinical care. Monitoring for adrenal insufficiency and brain involvement is crucial before symptoms arise. If neurological symptoms are detected early, stem cell transplantation can help slow down adrenoleukodystrophy’s progression. Additionally, doctors can focus on managing the symptoms and delaying the spread of the disease. Various anti-inflammatory and anti-seizure medications may be prescribed to alleviate the symptoms.
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Adrenoleukodystrophy Market Dynamics
Rare diseases, or RDs, affect only a small percentage of the population. Their prompt diagnosis and therapeutic management are challenging due to the scarcity of accurate epidemiological data and the limited availability of clinical information. The knowledge of the pathophysiology of many RDs is expanded in the recent few years owing to the rise in the adoption of new next-generation DNA sequencing technologies, which have also brought a new age of diagnostic and therapeutic research into this understudied health issue. As a result, diagnosing RDs became easier, sparking the need to develop new treatments.
The percentage of non-communicable diseases such as genetic disorders is rising in child mortality, morbidity, and disability. Common genetic disorders have been the center of policy and public health initiatives. Rare single-gene disorders significantly cause morbidity and premature mortality in suffering families. When taken together, they represent a significant public health cost often overlooked. As rare genetic diseases continue to rise, government authorities of several developed and developing countries are increasing various efforts to raise awareness and improve treatments. For instance, in May 2020, SwanBio Therapeutics announced that they had raised US$ 52 million to advance their viral-based gene therapy for adrenomyeloneuropathy, a form of X-linked adrenoleukodystrophy.
A strong gene and stem cell therapy pipeline is anticipated to drive market revenue growth. Gene therapy is being developed to enhance the treatment of multiple RDs, such as adrenoleukodystrophy. Numerous research centers and institutes focus on a wide range of therapeutics, which is projected to increase revenue production in the near future owing to its cost-effectiveness and efficiency. Following the FDA’s approval of the first gene therapy in 2017, the number of clinical trials for gene therapy has increased greatly. According to the American Society of Gene & Cell Therapy, 1,986 gene therapy products are in development. Furthermore, increased regulatory assistance presents the industry with an opportunity for growth. Thus, the development of new treatment strategies has a great impact on the adrenoleukodystrophy market.
However, the side effects of the medicines used to treat adrenoleukodystrophy are a major factor hindering revenue growth in the market. Lack of knowledge about treatment methods and prognosis restricts market revenue growth, as treatment is often ignored.
Adrenoleukodystrophy Market Ecosystem
The global adrenoleukodystrophy market is analyzed from four perspectives: disease type, treatment, end-user, and region.
Adrenoleukodystrophy Market by Disease Type
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Based on the disease type, the global adrenoleukodystrophy market is segmented as childhood cerebral adrenoleukodystrophy (ccALD), adrenomyelopathy, Addison’s disease, and others.
The childhood cerebral adrenoleukodystrophy (ccALD) segment accounted for the largest revenue share of the global adrenoleukodystrophy market in 2022. CALD is a rare and life-threatening neurological condition that affects newborns. Untreated patients have a high risk of passing away within five years of experiencing symptoms. The illness causes a rapid loss of neurological function after the initial onset of symptoms. The segment is growing rapidly due to an increase in the cases of adrenoleukodystrophy caused owing to rare genetic disorders in children. Adrenoleukodystrophy is estimated to affect 1 in 5,000 to 1 in 17,000 newborns (male and female, respectively) and approximately 1 in 20,000 to 1 in 30,000 newborn males. ccALD develops in approximately 40% of affected children and a smaller number of adult men.
Adrenoleukodystrophy Market by Treatment
Based on the treatment, the global adrenoleukodystrophy market is segmented into medications, gene therapy, bone marrow transplantation, and others.
The bone marrow transplantation segment accounted for the largest revenue share of the global adrenoleukodystrophy market in 2022. Endowment for stem cell research has expanded over the past few years due to the rising demand for novel treatment approaches in patients with rare genetic issues, such as adrenoleukodystrophy. The National Institute of Health has boosted its funding for stem cell research by more than 5% yearly, reaching its highest level in 2018. The market is experiencing growth due to the increasing interest of people in adopting alternative treatment options than the traditional ones. Using stem cells may offer various advantages to the patients, such as helping them avoid surgery and alleviating pain. It is a less intrusive therapy choice that encourages the body to mend itself using its own cells. Regenerative medicine helps to fasten the treatment for newborns due to its fast adaptability. Also, increased demand for personalized medicine is another major driver for segment revenue growth, as the usage of genetic data in clinical decision-making may rise due to new technology, advancing rare disease prevention, surveillance, and safer, more effective pharmacological therapy. Genetic testing, which promises to improve treatment choices for patients with genetic disorders, serves as the context for personalized medicine in this research. Genetically characterizing tumors and using personalized medicine can help develop new medications.
Adrenoleukodystrophy Market by End-user
Based on the end-user, the global adrenoleukodystrophy market is segmented into hospitals, specialty clinics, and others.
The hospitals segment accounted for the largest revenue share of the global adrenoleukodystrophy market in 2022. The market has assumed the lead as hospitals receive more financial support to invest in cutting-edge, innovative care. Hospitals are the best place to receive treatment as they have the necessary medications to provide patients with complete care. The main forces behind the segment development are the expanding access to healthcare reimbursements in many developed countries and the ongoing industrial advancements in regenerative therapeutics. The ease of the therapy process is facilitated by a team of doctors, surgeons, and other skilled healthcare professionals. In addition, the availability of sufficient funds allows hospitals to access advanced treatments and affordable diagnostic procedures. Therefore, patients can easily depend on hospitals for treatment regardless of their ability to pay.
Adrenoleukodystrophy Market by Region
Based on the region, the global adrenoleukodystrophy market is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa.
North America accounted for the largest revenue share of the global adrenoleukodystrophy market in 2022. North America presently dominates the market for adrenoleukodystrophy due to the presence of significant players. Additionally, the streamlined repayment regulations in North America and the increase in the prevalence of RDs are expected to support revenue growth in the region during the forecast period. Moreover, the presence of leading companies, availability of the latest advanced tools, a well-developed healthcare infrastructure, and payment coverage are considered to drive high demand for sophisticated healthcare services in the region. The market is also expanding dramatically through increased public awareness of RDs, such as adrenoleukodystrophy, their treatments, and related preventive measures. The increasing advancements in diagnostics of adrenoleukodystrophy are the major factor driving the market revenue growth. For instance, in September 2019, the Texas Department of State Health Services joined 15 other states to start NBS for adrenoleukodystrophy in every newborn. Further, the presence of several foundations, such as ALD Connect, to improve health outcomes for patients with ALD by empowering patients, raising awareness, and accelerating the translation of scientific advances into better clinical care, is fueling revenue growth in the North America adrenoleukodystrophy market.
The market in the Asia Pacific is also growing significantly owing to the high prevalence of adrenoleukodystrophy, the increase in research and development activities, and the rise in social awareness about the disease.
Adrenoleukodystrophy Market Competitive Landscape
The prominent players operating in the adrenoleukodystrophy market include:
Adrenoleukodystrophy Market Strategic Developments
Adrenoleukodystrophy (ALD) is a progressive, rare genetic disease that affects the nervous system’s adrenal glands, spinal cord, and white matter (myelin).
New treatment strategies and increased disease prevalence are the key trends fueling the global adrenoleukodystrophy market revenue growth.
High cost and lack of awareness about adrenoleukodystrophy may potentially restrict the revenue growth of the global market.
Bluebird Bio, Minoryx Therapeutics, S.L., MedDay Pharmaceuticals, Magenta Therapeutics, and Orpheris Inc are among the top market players.
The global adrenoleukodystrophy market is expected to grow at a revenue CAGR of 7.6% during the forecast period from 2023 to 2031.
*Insights on financial performance are subject to the availability of information in the public domain