FDA Grants 37 Breakthrough Designations in 2013

Since the FDA’s breakthrough therapy designation (BTD) was put into effect in July of 2012, pharma and biotech companies have certainly taken note, according to the new report, “Breakthrough Therapy Designation: A Breakthrough Year for Unmet Diseases,” from EP Vantage, the editorial arm of Evaluate. 

The FDA data indicate that no fewer than 141 BTDs have been applied for, of which 37 have been granted. Thus far, three breakthrough-designated products have received US approval: Roche's Gazyva, J&J/Pharmacyclics' Imbruvica and Gilead's Sovaldi.

The jury is still out, however, on what it actually means in practice for a project to be designated a breakthrough therapy, and attempts to highlight evidence of shortened approval times for BTD products look premature. The new EP Vantage report takes a deeper look at this new designation and how small and large pharma are looking to reap the benefits the designation is touted to provide.

“Although many companies have been quick to play up the potential advantages of a breakthrough designation, the fact is that no one actually knows what these are in practice,” said Jacob Plieth, report author and EP Vantage senior editorial analyst. “Little can be gleaned from the three products to have already received US approval, and many uncertainties still abound.”

A list of the 30 disclosed breakthrough projects reveals that the vast majority have favoured big pharma and big biotech — despite the fact that the FDA’s promise to provide assistance on clinical trial design and guidance on development was aimed at small, cash-strapped biotechs, notes Plieth. According to the report, the hope is that this trend will shift as the programme matures.

The report, based on market intelligence and analysis from EvaluatePharma can be downloaded here.