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Lysosomal Storage Disorder (LSD) Therapeutics Pipeline to Witness Remarkable Growth due to Increased Number of Pipeline Candidates by Drug Developers

According to a new research report Lysosomal Storage Disorder (LSD) Therapeutics – Pipeline Analysis 2019, Clinical Trials and Results, Patents, Designations, Collaborations, and Other Developments” published by Pharma Proff, LSD therapeutics currently exhibits a proliferating pipeline with 75+ therapeutic candidates.

LSD Therapeutics Pipeline Insights

LSD refers to a group of genetically inherited disorders, which are characterized by enzyme deficiencies. It results in accumulation of toxic chemical substances in the body’s cells. This group of disorders consists of approximately 50 diseases, including Metachromatic leukodystrophy; Mucolipidosis types I, II/III, and IV; Mucopolysaccharide storage disease; Gaucher disease; Niemann-Pick disease type C (NPC); Schindler disease types I and II; Tay Sachs disease; Pompe disease; and Sandhoff disease. These diseases affect different body parts, including heart, bones, brain, and central nervous system (CNS). Mental retardation, hepatomegaly, absurd growth of facial bones, and optic atrophy are some of the most common symptoms reported in the disease.

Genetic disorders are responsible for the occurrence of this disorder, in which most of the diseases are autosomal recessive in nature. Being gene related, genetic screening is a vital method for diagnosis of this disorder.

Insights into Pipeline Segments

According to the research findings, majority of pipeline drug candidates are being developed for intravenous route of administration. In this route of administration, entire administered dose reaches the systemic circulation immediately, which increases the bioavailability of drug.

Positive Clinical Trial Results are Expected to Drive LSD Therapeutics Pipeline

Companies that are involved in developing therapeutics for LSD have shown positive clinical results in various phases of drug development. For instance, in January 2019, Orphazyme A/S announced positive data of phase II/III trial of arimoclomol for the treatment of patients with NPC. The results of the study concluded that treatment with arimoclomol has a favorable safety and tolerability profile. The study also recorded efficacy of arimoclomol in lowering down the effects of the disease by 75%.

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Global Market for LSD Therapeutics is Expected to Increase Statistically in the next 10 years

Currently, Ceredase, Aldurazyme, and Fabrazyme by Sanofi-aventis Groupe, and Zavesca by Actelion Pharmaceuticals Ltd. are some of the U.S. Food and Drug Administration (USFDA) approved drugs for the treatment of LSD. Moreover, due to emergence of late- and mid-stage pipeline products in the market, the overall LSD therapeutics market is expected to grow significantly in the upcoming years.

Orchard Therapeutics Limited, Sanofi-aventis Groupe, Actelion Pharmaceuticals Ltd., Amicus Therapeutics Inc., Takeda Pharmaceutical Company Limited, Ultragenyx Pharmaceutical Inc., Valerion Therapeutics LLC, Actus Therapeutics Inc., Chiesi Farmaceutici S.p.A., Resverlogix Corp., AVROBIO Inc., Mallinckrodt LLC, CTD Holdings Inc., Orphazyme A/S, Idorsia Pharmaceuticals Ltd., and Protalix BioTherapeutics Inc. are some of the key players involved in the development of therapeutic drugs indicated for the treatment of LSD.

LSD Therapeutics Pipeline Analysis

  • By Phase
  • By Molecule Type
  • By Route of Administration
  • By Company

The report comprises detailed pipeline analysis of therapeutics being developed for the treatment of LSD. 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 LSD therapeutics development. Detailed regulatory approval procedures in the U.S., Europe, and Japan are also provided in this 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 LSD.