GSK-Amicus start late-stage trials of Fabry disease treatment
pharmafile | September 14, 2011 | News story | Research and Development | Amicus Therapeutics, Fabry disease, GSK
GlaxoSmithKline and Amicus Therapeutics are moving forward with their phase III programme for Fabry disease treatment Amigal.
The first patient has started dosing in a study comparing the safety and efficacy of Amigal (migalastat HCl) and enzyme replacement therapy (ERT) for the inherited condition.
While it affects only 5,000 to 10,000 people across the world, Fabry sufferers can experience pain, kidney failure and increased risk of heart attack.
GSK signed a development deal with Amicus for Amigal last October, which could net the US-based biopharmaceutical company up to $200 million depending on the oral drug’s performance.
Currently, there are two rivals: Shire’s Replagal and Genzyme’s Fabrazyme, both of which are subcutaneous ERTs.
But the distribution of Fabrazyme has been a recurring headache for Genzyme, now part of Sanofi, with the company apologising again this week for being forced to delay shipments.
The drug has been strictly rationed since its Allston Landing facility in Massachusetts was hit by viral contamination and other quality control problems in 2009.
Now the company is waiting for the FDA to clear its new manufacturing facility in Framingham, in Massachusetts, banking that once approved this will get supply of the product back on track.
“We believe Amigal has the potential to provide an important treatment option in Fabry disease,” said Philippe Monteyne, chief medical officer for GSK Rare Diseases.
The new trial, study 012, is a randomised, open-label, 18-month programme which the companies intend to run across 50 sites, enrolling 50 male and female Fabry patients currently receiving either Replagal or Fabrazyme.
Eligible patients will have a genetic mutation that may be addressable with Amigal. Those in the Amigal treatment arm will receive 150mg every other day, while those in the ERT alone arm will carry on their current regimen of ERT.
The primary outcome of efficacy will be renal function as measured by glomerular filtration rate (GFR) for both groups at 18 months. Secondary outcomes include renal function as measured by 24-hour urine protein.
This is the second of two phase III studies: already underway is study 011, a six-month, placebo-controlled study of Amigal at 37 sites worldwide.
“This is an important step in our clinical development plan and builds on the latest encouraging safety and renal function data from our ongoing phase II extension study,” said Amicus chief executive John Crowley.
The cause of Fabry disease is a deficiency of the enzyme alpha-galactosidase A (alpha-Gal A), which breaks down complex lipid globotriaosylceramide (GL-3).
Accumulation of GL-3 in tissues such as the kidneys, central nervous system, heart and skin is believed to cause Fabry’s various symptoms.
Adam Hill
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