Breakthrough year for unmet diseases, as FDA backs 37 designations in 2013

Since the US Food and Drug Administration’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, titled 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 (ROG: SIX) Gazyva (obinutuzumab), Johnson & Johnson (NYSE: JNJ)/Pharmacyclics' (Nasdaq: PCYC) Imbruvica (ibrutinib) and Gilead Sciences' (Nasdaq: GILD) Sovaldi (sofosbuvir).

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.