Fabry disease and Drug Development


Fabry disease is rare inherited enzyme deficiency disease and it is named after its discoverer  Johannes Fabry.  A deficiency of the enzyme alpha galactosidase A due to mutation causes a glycolipid known as globotriaosylceramide (abbreviated as Gb3, GL-3, or ceramide trihexoside) to accumulate within the blood vessels, other tissues, and organs. This accumulation leads to an impairment of their proper function. (Wikipedia)

There are two enzyme replacement therapies are currently available for treating Fabry disease, which are Agalsidase alpha (Replagal from Shire) and Agalsidase beta (Fabrazyme from Genzyme).

Recently, patient groups petitioned open licence Genzyme’s patents covering Fabrazyme due to ongoing shortage of drug supply. Another drug shire’s Replagal is not approved in USA.

GSK now licensed new drug for Fabry disease, which is migalastat HCl.

Image of Chemical Structure

Migalastat is first oral therapy for treating Fabry disease and pharmacologically this is alpha 2 galactosidase stimulant. Co-administration of Migalastat with Enzyme replacement therapy is being studied. Amicus Therapeutics would receive upfront payment of $30 million. In addition to the upfront payment, Amicus is eligible to receive about $170 million upon the successful achievement of development and commercialization milestones, as well as tiered double-digit royalties on global sales.

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About Guna

Interested to learn things around me
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2 Responses to Fabry disease and Drug Development

  1. Pingback: Amicus Study 011 In Fabry Disease Released Today ( $FOLD) « Translating Healthcare

  2. Pingback: Amicus Study 011 In Fabry Disease Released Today ( $FOLD) | Translating Healthcare

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