This approval is another example of the benefits of the Orphan Drug program, which provides incentives for the development of treatments for diseases affecting fewer than 200,000 patients in the United States a year. Until now, Pompe disease has had no approved treatment.
Until today, patients with diabetes who need insulin to manage their disease had only one way to treat their condition. It is our hope that the availability of inhaled insulin will offer patients more options to better control their blood sugars.