Lysosomal Storage Disorder (LSD) Therapeutics Pipeline Analysis

Posted by Manish Kumar on June 28th, 2019

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.

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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.

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.

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%.

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.

Some of the key players involved in the development of Lysosomal Storage Disorder therapeutics are 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., Orphazyme A/S, Idorsia Pharmaceuticals Ltd., AVROBIO Inc., Mallinckrodt LLC, CTD Holdings Inc., and Protalix BioTherapeutics Inc.