July 11, 2017
By Alex Keown, BioSpace.com Breaking News Staff
CRANBURY, N.J. – Shares of Amicus Therapeutics are skyrocketing this morning after the U.S. Food and Drug Administration (FDA) reversed course on the company’s Phase III treatment for Fabry disease. The change paves the way for Amicus to seek regulatory approval later this year.
John F. Crowley, chairman and chief executive officer of Amicus Therapeutics, announced the FDA’s change of heart this morning, which has caused the stock to spike more than 23 percent in the opening minutes of trading. Shares of Amicus are trading at $12.68 as of 9:39 a.m.
In November 2016, the FDA stunned Amicus when it called for a new clinical trial for its lead drug candidate migalastat, also known as Galafold in Europe, in order to win regulatory approval. The FDA was interested in additional safety data and sought a 35-patient trial, seeking additional data on gastrointestinal (GI) symptoms in Fabry patients who have an amenable mutation. That announcement was expected to delay the company by three years. However, with a new FDA commissioner and a new administration in the White House that has indicated a need for streamlining the regulatory process, Amicus is benefitting from the change.
Earlier this year on Rare Disease Day, President Donald Trump pointed to the work done by Crowley at his former company Novazyme Pharmaceuticals to develop a treatment for Pompe Disease. Crowley’s daughter Megan was diagnosed with the disease at 15 months old. On that day, Trump excoriated what he called the “slow and burdensome” approval process at the FDA that prevented treatments from getting into the hands of patients that could benefit.
Amicus said it will prepare a New Drug Application under Subpart H, which provides for accelerated approval. Amicus intends to base its NDA on existing data, including reduction in disease-causing substrate (GL-3), as well as the totality of data from completed clinical studies.
“This guidance from the FDA marks a tremendous step forward for thousands of people living with Fabry disease in the United States,” Crowley said in a statement. “We are moving ahead expeditiously with our NDA submission and accelerating the U.S. pathway for migalastat. 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.”
Jay Barth, Amicus’ chief medical officer, said company leadership believes it has a robust data package for the NDA.
“The data from our clinical trials, including the two largest pivotal studies ever completed in Fabry disease, have already supported approvals for migalastat in the EU, Israel and Switzerland, as well as our pending regulatory submissions in Japan, Canada and Australia. The FDA’s willingness to review migalastat data reflects what we believe is the gold standard in science-based, data-driven, patient-centric therapeutic development,” Barth said in a statement.
Migalastat is designed to bind with high affinity to the active sites of certain mutant forms of alpha-Gal A, the genotypes of which are referred to as amenable mutations, the company said. Fabry disease is a rare genetic disease caused by the lack of an enzyme that allows the body to break down lipids, which are fat-like substances that include oils, waxes and fatty acids. Without the ability to break down the enzyme, it can lead to kidney problems as well as heart attack or stroke. The disease affects about 10,000 people globally.There are an estimated 3,000 people in the U.S. are currently diagnosed with Fabry disease. The U.S. represents the single largest geography for Amicus to positively impact the lives of people with Fabry who have amenable mutations.
Migalastat was approved for use in Europe in May 2016. The drug competes with Shire ’s Replagel in the EU, However, the drug has a different mechanism for action, which means Galafold has the potential to be used alongside Shire’s drug.
