Adrenoleukodystrophy (ALD) is a rare genetic disorder linked with the X chromosome in males and females. Adrenoleukodystrophy (ALD) causes mutation of the ABCD1 gene in individuals which elevates ALD proteins. These proteins are used to clear toxic molecules known as saturated very long chain fatty acids. Thus accumulation of saturated very long chain fatty acid in tissue throughout the body leads to damage of myelin, thus causing neurological problems in patients.
Adrenoleukodystrophy (ALD) is most commonly observed in childhood and present in both males and females. Adrenoleukodystrophy (ALD) is further identified by its phenotypes such as childhood onset of ALD, Addison disease, adrenomyeloneuropathy and diagnosed with the help of blood testing, magnetic resonance imaging, vision screening, skin biopsy and fibroblast cell culture. Treatment used for Adrenoleukodystrophy (ALD) are stem cell transplant, adrenal insufficiency treatment, gene therapy and dietary therapy.
Global Adrenoleukodystrophy Drugs Market: Drivers
Adrenoleukodystrophy is a rare genetic disorder which is characterized by symptoms such as childhood onset of ALD, Addison disease, and adrenomyeloneuropathy. Therefore, depending upon symptoms, the type of an adrenoleukodystrophy drug have to be evaluated and treatment need to be prescribed accordingly. For instance, in May 2020, SwanBio Therapeutics, a developer of biological therapeutics, announced that they have raised US$ 52 million for advancement of their viral base gene therapy for adrenomyeloneuropathy. Funds were raised with the help of series A financing round. These factors are expected to drive growth of the global adrenoleukodystrophy drugs market.
Global Adrenoleukodystrophy Drugs Market: COVID-19 Impact Analysis
COVID-19 has affected several markets around the world. In case of global adrenoleukodystrophy drugs market, adrenoleukodystrophy patients are facing challenges due to reallocation of neurological intensive beds and neurological wards to COVID-19 patients. As a result, there is difficulty in management of emergencies related to neurological disorders. Furthermore, implementation of lockdowns and restrictions on transportation by governments of various countries have affected the supply chain activities, and led to a shortage in inventories of pharmaceutical companies.
Global Adrenoleukodystrophy Drugs Market: Restraints
Stem cell transplant is a treatment used for Adrenoleukodystrophy, but causes side effects such as interstitial pneumonia, liver damage and mucositis. Moreover, in adrenal insufficiency treatment, hydrocortisone is a major drug which affects cortisol levels in adrenoleukodystrophy patients and also weakens the immune system. Furthermore, gene therapy may lead to mutation of cells and inflammation due to wrong incorporation of DNA on DNA locations. These factors are expected to restrain the global adrenoleukodystrophy drugs market growth.
Global Adrenoleukodystrophy Drugs Market Regional Analysis
On basis of regions, the global Adrenoleukodystrophy market is segmented into North America, Latin America, Europe, Asia Pacific, Middle East, and Africa. In North America, pharmaceutical companies are focusing on advancement of therapies for treatment of adrenoleukodystrophy. For instance, in 2019, Magneta Therapeutics received approval for MGTA-456 regenerative medicine advance therapy designation for treatment of adrenoleukodystrophy from the U.S Food Drug and Administration.
In 2018, Europe PMC claimed that adrenal insufficiency is a life threating condition with mortality rate of 0.5 in 100 patients per year. Moreover, pharmaceutical companies are focusing on research and development of treatments for adrenoleukodystrophy to strengthen their share in the global adrenoleukodystrophy drugs market. For instance, in January 2020, Minoryx Therapeutics, a company specialised in innovation of drugs for central nervous system conditions, received an approval for lead drug candidate, leriglitazone (MIN-102), for treatment of all forms of x- adrenoleukodystrophy.
Global Adrenoleukodystrophy Drugs Market: Key Players
Key operating players in global adrenoleukodystrophy drugs market are Applied Genetic Technologies Corporation, MedDay SA, Bluebird bio, Inc., Minoryx Therapeutics S.L, ReceptoPharm Inc., Pfizer Inc., SOM Innovation Biotech, S.A, SwanBio Therapeutics and Viking Therapeutics, Inc.
On the Basis of Treatment, the global adrenoleukodystrophy drugs market is segmented into:
- Adrenal Insufficiency Treatment
- Gene Therapy Drugs
- Dietary Supplements
On the Basis of Patient Type, the global adrenoleukodystrophy drugs market is segmented into:
On the Basis of Distribution Channel, the global adrenoleukodystrophy drugs market is segmented into:
- Hospital Pharmacies
- Retail Pharmacies
- Online Pharmacies
On the Basis of Indication, the Global Adrenoleukodystrophy Drugs Market is segmented into:
- Childhood Adrenoleukodystrophy
- Addison Disease
On the basis of region, the global adrenoleukodystrophy drugs market is segmented into:
- North America
- Latin America
- Asia Pacific
- Middle East