Amicus Therapeutics' Fabry Treatment Shows Positive Results In Phase 3 Trials
By C. Rajan, contributing writer
Amicus Therapeutics has just announced the positive results from its second Phase 3 clinical trial of its new drug, Migalastat, to treat Fabry disease. Migalastat, Amicus' first main drug, is now closer to becoming the first oral treatment for patients with this potentially fatal genetic disorder.
In this study, Migalastat met both its co-primary end-points when compared against standard of care enzyme replacement therapies (ERTs), Sanofi's Fabrazyme and Shire's Replagal. The study showed that Migalastat was as effective as the ERTs over an 18-month period in 60 patients with a particular form of Fabry disease. It had a comparable effect to ERTs on kidney function, which was the main goal of the study.
John F. Crowley, Chairman and CEO of Amicus Therapeutics, stated, "We believe that this multi-year study unequivocally demonstrates that a Fabry patient on ERT with an amenable mutation can switch safely and effectively from ERT to Migalastat to treat their Fabry disease. These results clearly show that Migalastat is comparable to ERT in slowing the progression of Fabry disease and continues to demonstrate a favorable safety profile.”
Fabry disease is a potentially fatal inherited disorder caused by the deficiency of the alpha-Galactosidase A enzyme. The lack of this enzyme causes accumulation of fat in blood vessels throughout the body, leading to pain, kidney failure, heart attack and stroke. Fabry disease affects an estimated 1 in 40,000 persons and is more common in males than females.
Migalastat is tailored to treat 30-50 percent of Fabry patients with a specific cellular mutation, and is formulated for oral administration every other day, giving it a considerable advantage over the ERTs which require bi-weekly infusions.
An earlier Phase 3 study showed Migalastat significantly reduced fat build-up compared with a placebo after 12 months of treatment. With positive results from the two late-stage trials, Amicus has begun submitting data to the European regulators and plans to meet with the U.S. FDA later this year to discuss the drug's development.
The U.S, European Union and Japan have granted orphan drug status to Migalastat, which grants Amicus several incentives, including a period of marketing exclusivity.
Crowley said, “With every-other-day oral administration and a differentiated mechanism of action, Migalastat may offer significant advantages for patients without the need for bi-weekly infusions with ERT. Combined with our previous Phase 3 results, we have a compelling and consistent data set from both treatment-naïve and ERT experienced patients. Given these results and the great need for new and effective medicines, we plan to work with European and U.S. regulators to determine the fastest way to get Migalastat approved for all amenable Fabry patients."
Industry analysts expect the therapy to cost $200,000 per patient annually with estimated annual sales of about $400 million worldwide.