Fabry
disease is a type of inherited genetic disease caused due to abnormal
deposition of a fatty substance, called globotriaosylcera-mide, in blood vessel
walls throughout the body. This further leads to development of other
complications, which result in severe morbidities and can also be fatal, in
some cases. Anticipated growth of therapeutic pipeline of fabry disease can be
attributed to the rareness of the disease, as it is usually found in males at
an estimated ratio of one in 40,000 to 60,000 males. Although, the disorder
occurs in females as well, its prevalence is still unknown. This disease can be
cured using gene therapy and cell therapy, which are still in the development
phase. With increasing support from various funding agencies such as National
Organization of Rare Disorders (NORD), new and innovative therapies are being
developed to cure the problem. Therefore, the therapeutic pipeline of Fabry
disease is increasing.
Explore Report at: https://www.psmarketresearch.com/market-analysis/fabry-disease-therapeutics-pipeline-analysis
In
November 2016, FGK Clinical Research GmbH0 started Phase I, open-label,
multi-center study to evaluate pharmacokinetics, pharmacodynamics, and safety
of Moss-aGal in patients suffering from fabry disease. This study was completed
in February 2017. Moss-aGal is a recombinant human alpha-galactosidase that is
produced in moss. It is expected to be the first of its kind enzyme replacement
therapy (ERT) for patients with the genetic lysosomal storage disorder, which exhibits
optimized N-glycosylation patterns of the protein alpha-galactosidase A. In
July 2016, Ozmosis Research Inc. started a Phase I clinical study on autologous
stem cell transplantation of cluster of 34 positive (CD34+) cells engineered to
express Alpha-galactosidase-A in patients with fabry disease. This study is
expected to be completed in July 2022. In June 2016, Protalix Biotherapeutics
Inc., started a Phase III trial for testing safety and efficacy of PRX-102, as compared
to Agalsidase beta on renal function in patients with fabry disease previously
treated with Agalsidase beta. PRX 102 demonstrated improved circulatory
half-life, with better enzyme activity in target organs affected by fabry disease.
Some of the companies having a pipeline of fabry disease therapeutics include Greenovation Biotech GmbH, GlaxoSmithKline Plc, Protalix Biotherapeutics, Inc., Amicus Therapeutics, Inc, Shire Plc, Genzyme Corporation, Ozmosis Research Inc.
No comments:
Post a Comment