Shares of Amicus Therapeutics, Inc. (NASDAQ: FOLD) shot up 40% toward a 22-month high in active premarket trade Tuesday, after the biotechnology company said the Food and Drug Administration cleared the company to submit a new drug application for its Fabry disease treatment.
Last November, the FDA would not even consider approving migalastat, a treatment for the rare disease, until more data were collected, a process that was expected to take until 2019 or 2020.
Amicus says that the administration is reversing the decision, and that there is a clear path to approval. Amicus hopes to file a new drug application by the end of the year, and approval would take six months after that. That will shave three or four years off the drug's path to market if all goes well.
In Fabry disease, a substance called GL-3 builds up in the body, causing pain, kidney failure, heart disease and stroke. About 3,000 Americans have been diagnosed with the condition, according to the company. Migalastat is thought to work only in about half the patients with the disease, whose disorder is linked to certain genetic mutations.
"We are moving ahead expeditiously with our NDA submission and accelerating the U.S. pathway for migalastat," said Chief Executive John Crowley. "Today is a seminal moment in the development of migalastat and a testament to the dedication and perseverance of the patients, physicians and employees who have worked so hard on the development of this precision medicine."