According to a new research report “Adrenoleukodystrophy (ALD) Therapeutics Pipeline Analysis 2019, Clinical Trials and Results, Patents, Designations, Collaborations, and Other Developments” published by Pharma Proff, ALD currently exhibits a proliferating pipeline with 10 therapeutic candidates.
ALD Pipeline Insights
ALD is a X-linked genetic disease which falls under the family of leukodystrophies disorder. The disease is monogenetically inherited from parents to offspring, with 100% penetrance in men and 65% in heterozygous women. It is a multi-system disorder which prominently affects both central and peripheral nervous system, resulting in blindness, seizures and hyperactivity.
The pathophysiologic condition of disease includes the accumulation of saturated very long chain fatty acids (VLCFA) in serum and tissues of central nervous system, which is a major factor for immune system abnormality and demyelination of neurons. It mainly affects nervous system and adrenal glands and causes various adrenal and neurological problems, such as leg stiffness, muscles spasms and weakness, urinary problems, and sexual dysfunction.
Insights into Pipeline Segments
According to the research, many drugs acting as a therapeutic against ALD are administered orally. It has been found that oral route of administration is easy to use, non-invasive, and ensures high level of patient satisfaction.
Increasing Strategic Developments are Expected to Contribute to the Growth of the ALD Pipeline
Various companies are investing in the development of therapeutic drugs for ALD, and also tend to expand their product portfolio in near future. For instance, in 2015, Viking Therapeutics Inc. entered into research collaboration agreement with the U.S. based Kennedy Krieger Institute, amounting to $2.5 million. Under the terms of this agreement, Kennedy Krieger Institute’s scientist studied the Viking Therapeutics Inc.’s thyroid beta agonists, including VK0214 in the in- vivo model of X-ALD.
Surge in Designations Granted by Regulatory Bodies to Play a Pivotal Role in ALD Therapeutics Development
It has been observed that regulatory bodies are granting designations to the drugs in ALD pipeline, in order to pace up the development process. For instance, bluebird bio Inc. ‘s drug candidate, Lenti-D, received Orphan Drug Designation by the USFDA and EMA in 2012; and Breakthrough Therapy Designation by the USFDA in 2018. Similarly, Minoryx Therapeutics S.L. drug, MIN-102, received Orphan Drug Designation by the USFDA in 2017.
Browse report overview with detailed TOC on "Adrenoleukodystrophy (ALD) Therapeutics Pipeline Analysis 2019, Clinical Trials and Results, Patents, Designations, Collaborations, and Other Developments" at:https://www.pharmaproff.com/report/adrenoleukodystrophy
Some of the key players involved in the development of ALD are Poxel SA, bluebird bio Inc., Minoryx Therapeutics SL, NeuroVia Inc., SOM Innovation Biotech SL, Viking Therapeutics Inc., Orpheris Inc., MedDay Pharmaceuticals, Applied Genetic Technologies Corporation (AGTC), and Neuralgene.
ALD Therapeutics Pipeline Analysis
The report comprises detailed pipeline analysis of therapeutics being developed for the treatment of ALD. Comprehensive insights on the pipeline products have been provided, with special focus on strategic developments of key players, information on drug licensing, designations, financing, and grants, technological advancements, patents, and upcoming conferences. In addition, the report highlights the winning strategies of companies involved in the development of ALD therapeutics. Detailed regulatory approval procedures in the U.S., Europe, and Japan are also provided in the report. Furthermore, the report contains competitive analysis and extensive information on monotherapies, combination therapies, targets and mechanisms of action, and drug origin with respect to ALD.