In his speech to Congress last night, President Donald Trump called for changes to how the US Food and Drug Administration approves new drugs, the Hill reports.
"[O]ur slow and burdensome approval process at the Food and Drug Administration keeps too many advances… from reaching those in need," Trump said, according to a transcript of his remarks at the Wall Street Journal.
As an example, Trump described the case of Megan Crowley, who was diagnosed with Pompe disease when she was a little more than a year old. The rare glycogen storage disorder leads to decreased neuromuscular function, affects some 50,000 people worldwide, and can be fatal, FierceBiotech adds. Megan's dad, John Crowley, a former Bristol-Myers Squibb manager, founded a company called Novazyme after Megan, and then her brother, Patrick, were diagnosed that developed a recombinant alpha-glucosidase treatment for the disorder, David Kroll writes at Forbes. Genzyme has since acquired Novazyme, and Sanofi has since bought Genzyme, according to FierceBiotech.
"If we slash the restraints, not just at the FDA but across our government, then we will be blessed with far more miracles like Megan," Trump added.
But, FierceBiotech notes that the drug, the first for Pompe, was approved based on trials of just a few dozen patients and with a review time of less than a year. Indeed, Kroll points out that former FDA commissioner David Kessler tweeted that this example is not one of a "slow and burdensome" review process as it took nine months.
Kroll further adds that the safety evaluations uncovered that the enzyme therapy could cause serious allergic reactions in some people, leading the developers to change how it is dosed.
Additionally, FierceBiotech urges caution in interpreting what Trump's speech might mean for the drug approval process as it's unclear "just how literal he is being." Still, it adds that analysts see the speech as a broad positive for biotech.