NEW YORK (AP) -- Amicus Therapeutics Inc. said Monday that it won't be able to file for approval of its drug migalastat until more late-stage testing is completed.
Its shares fell by nearly 25 percent.
Amicus is studying migalastat as a treatment for a rare enzyme disorder called Fabry disease. The company has said the drug did not meet its main or secondary goals in a late-stage trial. The company is running an extension of that trial and has proposed doing additional statistical analysis. After a meeting with the Food and Drug Administration, Amicus said the FDA has decided the additional analysis is not enough to support a filing for marketing approval.
Migalastat is the company's most advanced experimental drug. Amicus does not have any products on the market.
Shares of the Cranbury, N.J., company fell 79 cents, or 24.8 percent, to $2.40 in midday trading. Earlier the stock hit an annual low of $2.30.
Amicus is currently running another late-stage trial of migalastat. It said it will continue its current and extension studies but does not plan to start any new trials.
Migalastat is designed to treat Fabry disease, an enzyme disorder caused by the buildup of a type of fat in the body's cells. The fat can cause pain and damage the kidneys and nervous system, among other problems.
Wedbush analyst Gregory Wade said the FDA seems to be more comfortable with the idea of reviewing migalastat based on a successful clinical trial instead of new analyses of a study that didn't meet its goals. He said some investors had hoped the agency would allow Amicus to apply for marketing approval based on new analyses, because the disease it treats is so rare.
Wade said the company will report more data from the first late-stage trial during the fourth quarter and will report results from the other late-stage study in the first half of 2014. He has an "Underperform" rating on Amicus shares because he did not expect a positive review for migalastat based on the data Amicus has reported. Wade added that he is unsure of the drug's commercial potential compared to other Fabry disease treatments.
- Health Care Industry
- Fabry disease