In his speech to Congress Tuesday night, President Donald Trump called the FDA drug approval process "slow and burdensome."
To support his argument, Trump pointed to Megan Crowley, a 20-year-old Pompe disease patient. Crowley was in the gallery of the House chamber listening to Trump's speech.
Here's what Trump said:
An incredible young woman is with us this evening who should serve as an inspiration to us all. Today is Rare Disease day, and joining us in the gallery is a Rare Disease Survivor, Megan Crowley. Megan was diagnosed with Pompe Disease, a rare and serious illness, when she was 15 months old. She was not expected to live past 5. On receiving this news, Megan's dad, John, fought with everything he had to save the life of his precious child. He founded a company to look for a cure, and helped develop the drug that saved Megan's life. Today she is 20 years old -- and a sophomore at Notre Dame.
Megan's story is about the unbounded power of a father's love for a daughter. But our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan's life, from reaching those in need. 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. In fact, our children will grow up in a Nation of miracles.
Absent specific policy details, Trump's speech leaves investors guessing about his real plans for the FDA and the drug approval process. He has not yet named an FDA commissioner.
Trump has previously expressed a desire to deregulate the FDA and lower the standards by which drugs are approved in the U.S. His comments about Megan Crowley seem to fit that theme.
But you could also argue Megan Crowley is an example of an FDA system that works well to quickly address the needs of patients suffering from rare diseases. Crowley's life was saved by Myozyme, an enzyme replacement therapy developed Genzyme, now a part of Sanofi (SNY) . Megan's father, John Crowley, formed a small company to develop Pompe disease drugs that was acquired by Genzyme and contributed to the successful development of Myozyme.
Myozyme was studied in two, uncontrolled clinical trials that enrolled a total of 39 Pompe disease patients. Genzyme submitted the Myozyme clinical data package to the FDA in July 2005. The FDA reviewed the drug's clinical data and approved it in April 2006 -- nine months later.
That hardly seems slow or burdensome.
The FDA has not been as kind to Amicus Therapeutics (FOLD) , the small orphan drug company run by John Crowley, Megan's father. John Crowley started Amicus after helping to develop Myozyme.
Last November, the FDA refused Amicus' request to submit an accelerated approval filing for Galafold, a new therapy to treat Fabry disease. The FDA told Amicus that an additional clinical study of Galafold would be required before the drug could be submitted and reviewed. Amicus estimates collecting the new data will take two years.
In contrast, European drug regulators approved Galafold in May 2016 using the same clinical data FDA deemed insufficient. Amicus is selling Galafold to Fabry patients in Europe but can't do the same in the U.S.
Critics of the current FDA approval process could point legitimately to John Crowley and Amicus -- more than his daughter Megan -- to support their argument for less regulation.
FDA supporters will counter that the agency's decision to turn away Galafold for more data did not harm Fabry patients in the U.S. or deny them treatment because Fabrazyme, an enzyme replacement therapy from Sanofi, is approved here and used to treat Fabry patients.
If Trump's FDA rhetoric becomes reality, Amicus might benefit. Will the rest of the biotech industry? Lowering approval standards at FDA is not something most biotech and drug industry executives want to see happen.