As per DelveInsight’s assessment, globally, about 3+ key pharma and biotech companies are working in the Cerebral Adrenoleukodystrophy (CALD) therapeutics landscape based on different Routes of Administration (RoA), Mechanism of Action (MoA), and molecule types. Several of the therapies are in the advanced stages of clinical development and are expected to launch in the coming years.
DelveInsight’s “Cerebral Adrenoleukodystrophy (CALD) Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Cerebral Adrenoleukodystrophy Market size, share, and trends in the seven major market (7MM) (i.e., the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan).
The Cerebral Adrenoleukodystrophy market report covers emerging drugs, current treatment practices, market share of the individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Cerebral Adrenoleukodystrophy (CALD): An Overview
Adrenoleukodystrophy (ALD) is an X-linked recessive genetic disorder caused by the abnormality in the ABCD1 gene present on the X chromosome that leads to the accumulation of very long-chain fatty acids (VLCFAs) in the brain, nervous system, and adrenal gland. The accumulation of VLCFAs leads to the deterioration of the myelin sheath, and without the sheath, neurons cannot conduct action potentials, which leads to the development of seizures and hyperactivity.
Cerebral Adrenoleukodystrophy (CALD) is the most severe form of Adrenoleukodystrophy (ALD), characterized by a breakdown of the protective sheath known as myelin, which surrounds and protects the nerve cells responsible for thinking and muscle control. CALD usually gets present in early childhood and progresses rapidly if untreated, leading to a severe loss of brain function and eventually death in most patients.
Cerebral Adrenoleukodystrophy (CALD) Market Key Facts
• In 2017, the total prevalent population of Adrenoleukodystrophy in the 7MM was 55,242.
• In Germany, a total of 1,895 diagnosed prevalent populations of ALD were found in 2017. Out of this diagnosed prevalent population, approximately 1,292 and 603 diagnosed prevalent populations of males and females were calculated, respectively.
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted Cerebral Adrenoleukodystrophy market size by analyzing the impact of current and emerging pipeline therapies. It also provides a thorough assessment of the market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the Cerebral Adrenoleukodystrophy market trend for each marketed drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MoA), Route of Administration (RoA), molecule types, competition with other therapies, brand value, and their impact on the market.
Cerebral Adrenoleukodystrophy (CALD) Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pool, future trends, and and views of key opinion leaders.
Cerebral Adrenoleukodystrophy (CALD) Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Cerebral Adrenoleukodystrophy market or expected to get launched during the study period. The analysis covers Cerebral Adrenoleukodystrophy market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Cerebral Adrenoleukodystrophy Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Key Developments in the Cerebral Adrenoleukodystrophy (CALD) Therapeutics Market
• On September 16, 2022, bluebird bio, Inc. (Nasdaq: BLUE) announced that the US FDA had granted accelerated approval for its gene therapy SKYSONA® (elivaldogene autotemcel), also known as eli-cel. SKYSONA® has generated positive results and slowed the progression of neurologic dysfunction in boys 4-17 years of age with early, active Cerebral Adrenoleukodystrophy (CALD). The approval of SKYSONA was based on data from bluebird bio’s Phase 2/3 study ALD-102 and Phase 3 ALD-104 study.
• In May 2022, SwanBio Therapeutics, a gene therapy company advancing adeno-associated virus (AAV)-based therapies, raised $56 million in second-round financing to advance its lead gene therapy candidate SBT101 for adrenomyeloneuropathy (AMN) into clinical testing. AMN is a form of X-linked adrenoleukodystrophy (ALD) that is caused by a deficiency in the ABCD1 gene.
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Cerebral Adrenoleukodystrophy (CALD) Therapeutics Analysis
There are approx. 3+ key companies which are developing therapies for Cerebral Adrenoleukodystrophy. Currently, Minoryx Therapeutics has its Cerebral Adrenoleukodystrophy drug candidates in the most advanced stage of clinical development.
Some of the key companies in the Cerebral Adrenoleukodystrophy (CALD) Therapeutic Market include:
• Minoryx Therapeutics
• POXEL SA
• Orpheris, Inc.
• bluebird bio, Inc.
• Minoryx Therapeutics
• Magenta Therapeutics
And many others
Cerebral Adrenoleukodystrophy (CALD) Emerging Drugs Covered in the report include:
• MIN-102 (Minoryx Therapeutics)
• PXL770 (POXEL SA)
And many more
Further Emerging Drugs & Key Companies are Covered in the Report. Download the Sample PDF to Learn More –
Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Cerebral Adrenoleukodystrophy Competitive Intelligence Analysis
4. Cerebral Adrenoleukodystrophy Market Overview at a Glance
5. Cerebral Adrenoleukodystrophy Disease Background and Overview
6. Cerebral Adrenoleukodystrophy Patient Journey
7. Cerebral Adrenoleukodystrophy Epidemiology and Patient Population (In US, EU5, and Japan)
8. Cerebral Adrenoleukodystrophy Treatment Algorithm, Current Treatment, and Medical Practices
9. Cerebral Adrenoleukodystrophy Unmet Needs
10. Key Endpoints of Cerebral Adrenoleukodystrophy Treatment
11. Cerebral Adrenoleukodystrophy Marketed Products
12. Cerebral Adrenoleukodystrophy Emerging Drugs and Latest Therapeutic Advances
13. Cerebral Adrenoleukodystrophy Seven Major Market Analysis
14. Attribute Analysis
15. Cerebral Adrenoleukodystrophy Market Outlook (In US, EU5, and Japan)
16. Cerebral Adrenoleukodystrophy Access and Reimbursement Overview
17. KOL Views on the Cerebral Adrenoleukodystrophy Market
18. Cerebral Adrenoleukodystrophy Market Drivers
19. Cerebral Adrenoleukodystrophy Market Barriers
21. DelveInsight Capabilities
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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