Lysosomal Storage Disorders | Pharmacor | G7 | 2014

The lysosomal storage disorders (LSDs) market will rapidly expand over our 2013-2023 study period, fueled primarily by an expansion in the number of LSDs with available therapies. A rich pipeline of novel agents is expected to launch, promoting sustained market growth. Among the LSDs expected to gain their first approved treatment are lysosomal acid lipase (LAL) deficiency, alpha-mannosidosis, and mucopolysaccharidosis VII (Sly syndrome). Additionally, currently treated LSDs also have novel drugs in the pipeline, aiming to improve on efficacy and convenience compared with existing therapies. Perhaps the biggest advance made by those therapies in the late-stage pipeline is the potential to address the neurological complications associated with LSDs. However, these emerging therapies will face major challenges, including safe and effective delivery to the central nervous system and identifying patients early enough for these drugs to provide therapeutic benefit. Nevertheless, as research continues to identify the potential causes of the various LSDs, the commercial opportunity to develop novel treatments will continue to grow.


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