Shire’s decision to ignore the US Fabry disease market triggered a ripple of anger among some patient advocates because it gave Sanofi a lock on the market, but Phase-III results from an experimental treatment by Cranbury, New Jersey’s Amicus Therapeutics could create a ripple of a different sort.
The company announced Wednesday results showing that its oral medication migalastat HCI worked as well as Sanofi’s Fabrazyme and Shire’s Replagal (both are approved injectables) among patients with what the drugmaker called “amenable mutations.”
Although Amicus describes migalastat as a “chaperone” drug and the company is exploring using the drug in tandem with other medications, this Phase-III study, called Study 012, assessed the drug as a standalone treatment. In addition to hinting at an oral alternative for these rare-disease patients, the Amicus small molecule stands apart from Fabrazyme and Replagal because unlike its marketed peers, it is not an enzyme-replacement therapy.
Phase-III results also indicate the drug could be used by patients who have already been diagnosed and started other therapies.
Fabry disease is a rare disease in which cells cannot break down and clear out certain fats. The National Institutes of Health says symptoms usually surface by adolescence and that the X-linked chromosome disease is associated with clouded corneas in male patients. Both male and female Fabry patients can suffer from problems including enlarged hearts, enlarged kidneys and gastrointestinal disorders.
Patients usually die early of stroke, heart attack or renal failure.