By Robin Respaut
(Reuters) – U.S. health regulators on Friday approved Amicus Therapeutics’ Galafold, the first oral therapy to treat Fabry disease, a rare, sometimes fatal condition in which accumulation of fat damages several organs.
Galafold, known chemically as migalastat, will be the first new Fabry treatment on the U.S. market in over 15 years, and will compete with Sanofi SA’s infused Fabrazyme.
The drug is already approved in seven other markets, including Europe, Japan, Australia and Canada.
Galafold is a long-term treatment for adult patients with specific genetic mutations. That accounts for about 35 percent to 50 percent of the 10,000 diagnosed Fabry patients worldwide. An estimated 3,000 people in the United States are diagnosed with the disease.
Amicus on Friday did not provide a U.S. price for Galafold. In an interview prior the FDA’s approval, Chief Executive John Crowley said the company was committed to keeping new drugs priced at parity with or slightly below existing treatments, rather than charging a premium.
“If we’re going to have more medicines for people living with rare diseases, we’ve got to bend the cost curve,” Crowley said.
In Europe, where each country negotiates drug pricing, Galafold costs between $200,000 and $250,000 annually. In Japan, it is slightly higher, Crowley said.
Amicus said it would not raise the U.S. price of Galafold higher than the rate of consumer inflation, and has said it would reinvest a portion of the drug’s sales to develop new Fabry therapies.
“When we’re involved in making a medicine for your disease, we’re involved in it with you forever until there is a cure,” said Crowley, who first ventured into the biotechnology field to seek a treatment for two of his children diagnosed with the rare condition known as Pompe disease.
In the second half of 2018, Crowley said he expects Amicus to launch “a number of gene therapy programs.”
Amicus shares were up 4.2 percent following the FDA approval.
Fabry disease is caused by a deficiency of the enzyme alpha-galactosidase A, creating a fatty buildup in the organs that can cause kidney failure, heart disease or stroke.
A pill taken every other day, Galafold binds to the enzyme to help it break up the fat.
Sanofi’s Fabrazyme, by contrast, infuses replacement enzymes into the body. A second enzyme replacement therapy, Shire’s Replagal, is not available in the United States.
The U.S. approval represents a reversal of fortune for New Jersey-based Amicus, after the FDA nearly two years ago told the company it needed additional clinical data on Galafold’s gastrointestinal impact, which could have delayed its U.S. entry by years.
But in July 2017, the FDA said it would review the therapy without the additional data, one of several turnarounds by the agency following guidance of the 21st Century Cures Act, which aims to speed novel drugs to market.
The FDA shift came a few months after President Donald Trump, in an address to Congress, recognized Crowley’s daughter, Megan, then age 20, who suffers from Pompe disease. Trump criticized the government’s “slow and burdensome approval process” that “keeps too many advances, like the one that saved Megan’s life, from reaching those in need.”
Wall Street is forecasting Galafold sales of nearly $90 million this year, climbing to $171 million in 2019, according to Thomson Reuters data.
Fabrazyme recorded sales of 722 million euros ($838 million) in 2017, of which 51 percent came from the United States. Replagal sales reached $472 million in fiscal 2017.
(Reporting by Robin Respaut; editing by Bill Berkrot, Michele Gershberg, Phil Berlowitz, Jonathan Oatis and Diane Craft)