The U.S. Food and Drug Administration (FDA) has advised Sangamo Therapeutics that positive results from a single well-controlled clinical trial, in addition to existing evidence, may be sufficient to form the basis for approving its Fabry disease gene therapy candidate. For Sangamo, that means that one…

In 2003, when I attended the U.S. Food and Drug Administration (FDA) meeting that would determine whether to approve Fabrazyme (agalsidase beta), which became the first approved treatment developed specifically for Fabry disease in the U.S., I was given an opportunity to make comments to the decision-making panel. Having…

The U.S. Food and Drug Administration (FDA) has designated FLT190, an investigational gene therapy for Fabry disease,  an orphan drug to support and speed its development and possible review, Freeline Therapeutics, its developer, announced.

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

The U.S. Food and Drug Administration has granted accelerated approval to Galafold (migalastat) 123 mg capsules to treat adults with a confirmed diagnosis of Fabry disease who have an amenable galactosidase alpha gene (GLA) mutation. “This FDA approval of Galafold is a transformative moment for people in the U.S. living…