Growing
incidence of the pompe disease globally projected to drive the sales of the
pompe disease therapeutics over the forecast period.
According to a research study,
approximately 5,000 to 10,000 people are affected with pompe disease around the world. Moreover, according to National Organization for Rare Disorders, pompe disease incidence is generally placed at approximately 1 in 40,000 births in the United States However, the incidence of pompe
disease is much less common in Holland and Australia with an incidence of 1 in
145,000. In addition, It is even rarer in Portugal,
with one in
600,000 newborns diagnosed with the pompe disease Among
the all disease types, incidence rate of infantile pompe disease is less common
compared with late-onset pompe disease. According to a research study, incidence of infantile-onset Pompe disease is estimated at one in138,000 people in the Netherlands. However, one in
57,000 adults has late-onset Pompe disease in the country.
Market
players focus on the development of innovative products to treat pompe disease. Currently, there are few molecules currently undergoing various stages
of clinical trials for the treatment of pompe diseases. For instance,
Sanofi Genzyme developing neoGAA,
an enzyme replacement therapy molecule for the treatment of infantile and late onset pompe disease and the molecule is under phase 3 clinical trials Amicus therapeutics developing
AT-GAA (ATB200/AT2221), is a novel dual-mechanism therapy under development
currently entered into phase III studies. It combines 2 investigational agents
such as ATB200 is an investigational next-generation recombinant human GAA
(rhGAA) intravenous ERT designed with optimized glycosylation and high levels
of mannose 6-phosphate residues for better uptake in target tissues. AT2221 is
an orally administered pharmacologic chaperone given prior to infusion of
ATB200 to stabilize ERT in blood.
Furthermore,
companies actively developing biologic drugs to provide better treatment options for pompe disease patients by conducting multiple clinical trials and
head-on-head trials with existing drugs.
In October 2015, Audentes
Therapeutics announced that, company has raised $65 Million In Series C
financing for development and commercialization of
gene therapy products for patients with serious, rare diseases In addition in 2018, Actus
Therapeutics filed IND for AAV2/8-LSPhGAA with funding from the National
Institutes of Health and initiated the clinical trials for the treatment of
pompe disease
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