Boehringer Ingelheim’s nintedanib has been awarded breakthrough therapy designation by the US Food and Drug Administration as a treatment for idiopathic pulmonary fibrosis.

IPF, a serious and life-threatening disease that causes permanent scarring of the lungs, affects as many as 132,000 Americans and there are currently no FDA-approved treatments. However, that situation is likely to change in the near future given that nindetanib was filed earlier this month with the agency, five weeks after InterMune’s rival IPF product Esbriet (pirfenidone) was resubmitted to the FDA, having been rejected in May 2010.

Sabine Luik, head of medicine and regulatory affairs at Boehringer’s US pharmaceuticals unit, said that getting breakthrough designation “will help expedite its development and review as a potential treatment option for patients with IPF”. The tyrosine kinase inhibitor has already been granted a priority review (in June 2014) and fast-track designation a year earlier.

Questions about BTD validity

However, the fact that nintedanib has been granted BTD when it is so far down the regulatory line has led to questions about the validity of the whole process established by the FDA in 2012. It is intended to expedite drugs for serious or life-threatening conditions where preliminary clinical evidence indicates that the therapy may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

The FDA clearly has more than preliminary evidence to look at with nintedanib  and some commentators are calling into question the point of having another level of speedy review.

A spokesperson for Boehringer told PharmaTimes that “we cannot speculate on how the BTD will impact approval”.