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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Growth Rate |
CAGR of 7.6% from 2023 to 2031 |
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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.
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 because the bodies are unable to break down these long-chain fatty acids. The fatty membrane (myelin) that protects nerves in the brain and spinal cord 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.
Also, changes in cognition and conduct are frequent adrenoleukodystrophy symptoms. Men are more prone to adrenoleukodystrophy than women. Treatment aims to reduce or halt the disease's symptoms and progression. Adrenoleukodystrophy has no known treatment. With the recent implementation of newborn screening (NBS) for adrenoleukodystrophy, the disease can now be identified before it manifests clinically, providing a chance for further phenotyping and genetic characterization. A promising new therapy for childhood cerebral adrenoleukodystrophy is developing that combines gene therapy and autologous hematopoietic stem cell transplantation. The clinical management and outcomes of adrenoleukodystrophy are beginning to change as a result of these advancements in adrenoleukodystrophy screening and therapy, but there are still many unknowns, including the best time to start treatment and clinical care for women with adrenoleukodystrophy as well as clinical monitoring for adrenal insufficiency and brain involvement before clinical manifestations. However, suppose the diagnosis is performed as soon as neurological symptoms begin to appear. In that case, stem cell transplantation may limit the progression of adrenoleukodystrophy. Further, doctors may concentrate on treating the symptoms and delaying the spread of the illness. Several anti-inflammatory and anti-seizure drugs are administered to relieve symptoms.
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Market Dynamics
Compared to other diseases, rare diseases (RDs) are clinical conditions that only impact a small percentage of the general 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 patho-physiology of many rare diseases is expanded owing to 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, the diagnosis of rare disease became easier, sparking the need to develop new treatments. Also, the percentage of non-communicable diseases like genetic disorders in child mortality, morbidity, and disability is rising. Common genetic disorders have been the center of policy and public health initiatives. For afflicted families, rare single gene disorders significantly cause morbidity and premature mortality. When taken together, they represent a significant public health cost that is frequently overlooked. Due to the increasing prevalence of rare genetic diseases, several initiatives are taken to increase awareness and improve treatments. For instance, in May 2020, SwanBio Therapeutics announced that they have raised the fund of US$ 52 million for the advancement of their viral base gene therapy for adrenomyeloneuropathy, a form of X-linked adrenoleukodystrophy.
Moreover, a strong gene therapy and stem cell therapy pipeline is anticipated to drive market growth. Gene therapy is being developed to enhance the treatment of multiple rare diseases like adrenoleukodystrophy. Numerous research centers and institutes focus on a wide range of therapeutics, which is anticipated 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 greatly increased over years. The American Society of Gene & Cell Therapy (ASGCT) estimates that 1,986 gene therapy products are currently being developed. Additionally, stronger regulatory support offers the industry a chance to expand. 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 is a major factor hindering the growth of adrenoleukodystrophy market. Additionally, the lack of awareness about the newly developed treatment strategies and the diseases outlook most of the time the treatment remains overlooked which puts a cap on the market growth.
Market Ecosystem
The global adrenoleukodystrophy market is analyzed from three perspectives: diseases 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. The neurologic condition is known as cerebral adrenoleukodystrophy, or CALD, a life-threatening rare adrenoleukodystrophy which affects newborns. Nearly half of the patients who do not receive treatment pass away within five years of the onset of symptoms. The illness causes a rapid loss of neurological function after the initial onset of symptoms. The rising prevalence of adrenoleukodystrophy is the crucial reason for the fast growth of this segment owing to the increased occurrence of rare genetic disorders in children. Adrenoleukodystrophy is estimated to affect 1 in 5,000 to 1 in 17,000 newborns (both male and female), and approximately 1 in 20,000 to 1 in 30,000 newborn males. Cerebral adrenoleukodystrophy develops in approximately 40% of affected children and in a smaller number of adult men.
Adrenoleukodystrophy Market by Treatment
Based on the treatments, 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 like 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's growth is fueled by rising interest in treatment options other than the traditional ones. The patient may benefit from stem cells to prevent surgery and lessen 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 newborn because of its fast adaptability. Also, increased demand for personalized medicine is another major driver for the market as the use 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. The genetic characterization of tumors and personalized medicine can both guide the creation of new medications.
Adrenoleukodystrophy Market by End-user
Based on the end-users, the global adrenoleukodystrophy market is segmented as hospitals, speciality 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. Because they have the necessary medications to provide patients with complete care, hospitals are the best place to receive treatment. The main forces behind the segment development are the expanding access to healthcare reimbursements in many developed countries and ongoing industrial advancements in regenerative therapeutics. The ease of the therapy process is facilitated by a team of doctors, surgeons, and other specialists on staff. Additionally, having enough money gives hospitals access to cutting-edge, recently introduced treatment choices and affordable diagnostics. 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 of North America and the increasing prevalence of rare diseases are expected to support the growth. Moreover, the presence of industry titans, along with the newly launched advanced tools, a well-developed healthcare infrastructure, and payment coverage, are considered to drive high demand for sophisticated healthcare services in the area. The market is also expanding dramatically through increased public awareness of rare diseases such as adrenoleukodystrophy, their treatments, and related preventive measures.
Additionally, the increasing advancement in diagnostics of adrenoleukodystrophy is the major factor driving the market growth. For instance, in September 2019, Texas Department of State Health Services joined 15 other states to start newborn screening for adrenoleukodystrophy in every newborn. Further, the presence of several foundations such as, ALD connect, to improve health outcomes for patients with adrenoleukodystrophy (ALD) by empowering patients, raising awareness, and accelerating the translation of scientific advances into better clinical care are driving the growth of adrenoleukodystrophy market in this region.
On the other hand, the market in the Asia Pacific region is also growing significantly owing to the high prevalence of adrenoleukodystrophy and increasing research and development as well as social awareness.
Competitive Landscape
Adrenoleukodystrophy has a moderately competitive global market due to new product introductions, mergers, and acquisitions. The introduction of novel therapeutics, obtaining approval for those products, and participating in other activities like awareness programs are some key development strategies that many organizations emphasize. Additionally, the market classified partnerships, acquisitions, and other collaborations as improved growth strategies. Given the rising global market demand for adrenoleukodystrophy, these steps have increased the likelihood of having profitable growth prospects in the future. Notable market participants include,
Strategic Development
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 to fuel the growth of the global adrenoleukodystrophy market growth.
High cost and lack of awareness about adrenoleukodystrophy are potentially restricting the growth of the global market.
Key companies operating the global adrenoleukodystrophy market are Bluebird Bio, Minoryx Therapeutics, S.L., MedDay Pharmaceuticals, Magenta Therapeutics, Orpheris Inc, SOM Biotech, NeuroVia, Inc..
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