President Donald Trump in his inaugural speech to a joint session of Congress characterized the approval process of drugs at the U.S. Food and Drug Administration as burdensome and slow. Trump used the example of the now 20-year-old Megan Crowley who was diagnosed with a rare disease known as Pompe Disease at the age of 15 months. Doctors gave her less than 5 years to live but her father, John Crowley, started a firm with the aim of getting a cure for the disease. In Trump’s view the exception that the FDA made to this case and which allowed a faster approval process should be the norm.
“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,” said Trump in his inaugural speech to a joint session of the U.S. Congress.
Easier standards
To quicken the process of approval at the FDA, Trump has hinted previously that the way to go was to ease standards and further deregulation.
Genzyme, the company that her father founded to save her life developed a therapy for enzyme replacement known as Myozyme. To undertake trials for Myozyme, 39 patients who had Pompe disease were recruited for two clinical trials which were uncontrolled. After the trials, the package containing the clinical data was submitted to the Food and Drug Administration in July of 2005. Following the review of the clinical data, the FDA approved the drug 9 months later.
More data
Genzyme was later acquired by Sanofi and John Crowley went on to form another company named Amicus Therapeutics, Inc. (NASDAQ:FOLD). The latter company cannot, however, claim the same success with the FDA though as a request for accelerated approval for a new drug for treating Fabry disease was rejected since more clinical data was needed. While this could be used in the argument calling for a faster approval process, it is important to note that the disease already has a drug from Sanofi that is already available unlike in the case of Pompe where no cure previously existed.
Amicus Therapeutics Inc gained 17.10% on Wednesday to close the day at $7.60.