GSK signs rare diseases deal with Amicus
pharmafile | November 1, 2010 | News story | Research and Development, Sales and Marketing | Amicus Therapeutics, Fabrazyme, Fabry disease, GSK, GlaxoSmithKline, pharmacological chaperones
GlaxoSmithKline is partnering with US biopharmaceutical company Amicus Therapeutics to develop and commercialise its late-stage Fabry disease drug Amigal.
GSK will make a $30 million upfront license payment to Amicus, and the firm will be eligible for milestone payments of around $170 million and tiered double-digit royalties on global sales of the drug.
GSK will also take a $31 million stake in the company as part of the deal for Amigal (migalastat HCl), which is currently in phase III trials for the rare inherited disorder Fabry disease.
Marc Dunoyer, global head of GSK rare diseases, said: “This strategic collaboration is another significant milestone in delivering our vision for GSK rare diseases.
“Our focus now is to continue to advance Amigal for Fabry disease and it is our hope to deliver a first-in-class, oral medicine to the thousands of people worldwide living with this devastating rare disease.”
Currently, there are two treatments available for Fabry disease: Replagal from UK biotech firm Shire and Fabrazyme from US-based Genzyme.
In July the CHMP advised doctors to switch patients from Fabrazyme to Replagal due to Genzyme’s ongoing manufacturing woes.
Both are subcutaneous enzyme replacement therapies that aim to reduce the effects of Fabry disease.
Fabry disease is an inherited disorder where the patient cannot break down a complex lipid called globotriaosylceramide (GL-3). The accumulation of GL-3 is believed to cause the various symptoms of Fabry disease, including pain, kidney failure, and increased risk of heart attack and stroke.
Amigal is an investigational treatment with the potential to be the first in a new class of oral, small molecule medicines called pharmacological chaperones.
It is designed to selectively bind to and stabilise the target enzyme α-galactosidase A (α-Gal A), that helps proper trafficking of the enzyme to the lysosomes, where it is needed to break down the target substrate globotriaosylceramide (GL-3).
Recent phase II studies of Amigal showed that in subjects identified as responders to Amigal, the drug led to increased levels of α-Gal A, reduced levels of GL-3 as measured in renal interstitial capillary cells from kidney biopsies and in urine, and a potential positive impact on renal function.
It is currently estimated that Fabry disease affects approximately 5,000 to 10,000 people worldwide.
In October GSK partnered with one of the biggest biomedical charities in Italy and the country’s largest private research institute to develop novel treatments for rare genetic disorders.
Ben Adams
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