(Reuters) - The European Medicines Agency recommended approving American biotech drug developer Amicus Therapeutics Inc’s experimental Fabry disease treatment.
The European health agency said on Friday that migalastat, the drug to treat the inherited disease, is being recommended for adults and adolescents aged 16 years and older. (bit.ly/25APktn)
The recommendation came after a roller-coaster run for migalastat. A year after a late-stage study failed to meet its main goal in 2012, GlaxoSmithKline Plc had returned the rights to migalastat and dissolved its stake in the company.
Cranbury, New Jersey-based Amicus said in February that it expects to meet with the U.S. Food and Drug Administration in the second quarter to submit a new market application for migalastat after the regulator in October asked for a more comprehensive analysis of trial data.
However, analysts do not see the European regulator’s recommendation to have an impact on the FDA’s decision.
“Although the bar is still higher in the U.S., this news will be taken positively and help restore investor confidence in management execution,” Leerink Partners analysts wrote in a note.
The biotechnology company lost about $956 million in market capitalization on Oct. 2 after it said it would delay filing for U.S. marketing rights for its lead drug, migalastat.
Shares of Amicus, which had a market value of about $1 billion as of Thursday’s close, were up 3 percent at $8.71 in late trading on Friday.
The company is hoping to make migalastat the first oral treatment for patients with a form of Fabry disease, a potentially fatal disorder that affects about 1 in 40,000 to 60,000 men and occurs less frequently in women.
Fabry disease is caused by the build up of a type of fat, most notably in the kidneys, due to the deficiency of the alpha-Gal A enzyme. The accumulation damages cells and could lead to kidney failure, heart attacks and strokes.
Amicus said on Friday that it expected final decision from the European Commision in the second quarter.
Reporting by Vidya L Nathan and Rahul B in Bengaluru; Editing by Maju Samuel
Our Standards: The Thomson Reuters Trust Principles.