Amicus's lead drug nears approval as late-stage trial succeeds
(Reuters) - Amicus Therapeutics Inc moved a step closer to bringing its first drug to the market after trial results paved the way for its lead drug to become the first oral treatment for patients with a disorder that causes an abnormal build-up of fat.
The drug developer's stock jumped 20 percent to $5.47 in early trading on Wednesday after the results from the second late-stage trial were released.
Amicus has started submitting trial data to the European health regulator and plans to meet the U.S. Food and Drug Administration later this year to advance the development of the drug, the company said in a conference call.
Regulatory authorities in the United States, the European Union and Japan have granted migalastat orphan drug status, providing Amicus certain incentives including a period of marketing exclusivity.
The drug has already been proved to significantly reduce the abnormal accumulation of fat, compared with a placebo, after 12 months of treatment in an previous late-stage study.
Fabry disease is a potentially fatal inherited disorder characterized by the buildup of a particular type of fat, most notably in the kidneys, caused by the deficiency of an enzyme called alpha-Gal A.
This progressive lipid accumulation results in cell damage, leading to pain, hearing loss, kidney failure, heart attack and stroke.
As a monotherapy, migalastat works by binding to the alpha-Gal A enzyme and helping it break down the lipids. It is tailored to treat 30 percent to 50 percent of Fabry patients who carry a specific cellular mutation.
Migalastat had a comparable effect to ERTs on the kidney function of patients - the main goal of the study, the company said.
The drug needs to be orally administered every other day, giving it an edge over standard treatments such as Sanofi SA's Fabrazyme and Shire Plc's Replagal as these ERTs require bi-weekly infusions.
GlaxoSmithKline returned the rights to migalastat to Amicus last November, a few months after drug failed to show statistically significant reduction in kidney lipid levels at 6 months.
Amicus is also testing migalastat, in combination with the current treatments for the disease, for Fabry patients who are not amenable to the oral formulation of the drug.
Up to Tuesday's close of $4.57, the Cranbury, New Jersey-based company's stock had risen about 148 percent since the results of the earlier study were released on April 29.
(Reporting by Natalie Grover in Bangalore; Editing by Savio D'Souza)