In his first speech to a joint session of Congress on Tuesday, President Donald Trump drew attention to 20-year-old Megan Crowley, who was diagnosed with a deadly muscle-weakening condition called Pompe disease when she was hardly a year old.
Megan's father, John Crowley, is today the CEO of Amicus Therapeutics, a biotechnology company that specializes in therapies for rare diseases. The company has a pipeline of therapies for a range of human genetic diseases, including Pompe disease, according to Amicus' website.
CEO Crowley sought to find a cure for his daughter's rare illness — becoming a pioneer for the drug industry — and Megan's life was saved as a result of his hard work.
His mission is to "champion the development of new medicine," Crowley said in an interview on CNBC's "Power Lunch" on Wednesday.
And in working to grow Amicus' pipeline of products, Crowley's team must now work with the Trump administration. President Trump has said he wants to make it easier for pharmaceutical companies to win regulatory approval for their drugs, and he also wants to bring prices down.
The pharmaceutical industry has been watching Trump closely, digesting what he has to say, as it could impact future research and development in this space.
Recently, the Food and Drug Administration refused Amicus' request for accelerated approval of a drug, but Crowley told CNBC he is committed to working with the organization. "We have a great relationship with the FDA, and we're committed to work through issues and evaluate what is in the best interest of the patients."
There are more than 7,000 rare diseases, impacting more than 30 million people globally, Crowley said. Putting patients first is number one, he said, and his team's work also must be driven by science.
Crowley expressed his optimism, following Trump's remarks Wednesday, that the president would be "constructive" for the industry and for regulators moving forward.
On the heels of the company's full-year results out Wednesday morning, shares of Amicus closed the day up more than 17 percent. The stock has gained more than 52 percent for the year.