Which therapy areas virtually guarantee a company US approval on first review? If oncology is by far the most obvious answer, musculoskeletal indications, including rheumatoid arthritis, gout and certain rare disorders, might come as a bigger surprise.
Statistics from the FDA’s Center for Drug Evaluation and Research (CDER) for 2011-17, supplemented by EvaluatePharma data, show that drugs against these two disease areas that secured US approval did so on the first review cycle 100% of the time. At the other end of the scale, immunomodulators only managed a 57% success rate in this period (see charts below).
The big caveat of this analysis is that it looks only at drugs that actually got approved. Inclusion of still unapproved disappointments like Lilly/Incyte’s Olumiant – recently re-reviewed after a complete response letter – and PTC’s Translarna (slapped with a refuse-to-file letter) would drag down the success rates for the musculoskeletal category.
Musculoskeletal approvals’ first-cycle approval rate comes courtesy of drugs like Xeljanz, Otezla and Exondys 51.
Meanwhile, the immunomodulator segment was dragged down by Firazyr, Nulogix and Granix, though the second-cycle approvals of these three drugs took place over five years ago. More recently, first-cycle approval of Sanofi/Regeneron’s all-important dermatitis drug, Dupixent, helped restore the balance.
It should also be noted that the dominance of oncology is twofold: not only does this therapy area represent the category most likely to get the FDA’s blessing on the first time of asking, more cancer drugs – 66 – than any others have been approved by the CDER during the timeframe in question.
The years 2013-15 were dominated by first-cycle CDER green lights for such oncology drugs as Imbruvica, Gazyva, Blincyto, Keytruda, Opdivo, Alecensa and Tagrisso; on the flip side this period finally saw Sprout’s Addyi and Merck & Co’s long-suffering Bridion limp over the finish line – at the third and fourth time of asking, respectively.
Bridion is an intensive care anaesthetic that was a hangover from Merck’s takeout of Schering-Plough, and took seven years from first being filed to make it to approval. Only one other drug during this time period fared worse: in 2012 the FDA finally approved Discovery Laboratories’ Surfaxin for respiratory distress in pre-term babies, after almost eight years, five times of asking and the scalp of one chief executive.
Other therapy areas prone to suffering rejection at first time of asking include respiratory, endocrine and haematology. Still, as even the makers of Bridion and Surfaxin might argue, it is better to come to the party late than never.