Regulatory Focus™ > News Articles > First-in-Class, Rare Disease Drug Approvals Dominated in 2015: A Breakdown from FDA

First-in-Class, Rare Disease Drug Approvals Dominated in 2015: A Breakdown from FDA

Posted 26 January 2016 | By Zachary Brennan 

First-in-Class, Rare Disease Drug Approvals Dominated in 2015: A Breakdown from FDA

Last year was a banner year for the US Food and Drug Administration (FDA) in terms of new drug approvals, with many of the new drugs coming to market to help rare disease patients and offer new first-in-class options.

The total of 45 approvals was well ahead of FDA’s average of about 28 novel approvals between 2006 and 2014, and 36% of those new approvals were for first-in-class drugs while almost half (47%) were to treat rare diseases, or ailments that impact 200,000 or fewer Americans.

And though the agency patted itself on the back for its efficient work in approving new therapies, FDA was also quick to praise the pharmaceutical industry’s work in developing more high-quality drugs.

“More important than the quantity of novel drugs approved in 2015 are the qualities of the novel drugs the pharmaceutical industry has developed and the important new roles these drugs are serving to advance medical care,” FDA said in a new report.


FDA highlighted noteworthy first-in-class products that were recently approved, including: Merck’s Bridion (sugammadex), to reverse post-surgical neuromuscular blockade caused by certain kinds of anesthesia; Pfizer’s Ibrance (palbociclib), to treat advanced (metastatic) breast cancer and Boehringer Ingelheim’s Praxbind (idarucizumab), to reverse adverse anticoagulant effects caused by the blood-thinner drug Pradaxa (dabigatran).

As for noteworthy examples of rare disease drugs approved in 2015, FDA points to Alexion’s Kanuma (sebelipase alfa) to treat lysosomal acid lipase deficiency, a rare inherited genetic disorder and Strensiq (asfotase alfa), a long-term enzyme replacement therapy in patients with a serious and sometimes fatal bone disease, as well as Vertex’s Orkambi (lumacaftor/ivacaftor), a cystic fibrosis therapy.

Oncology drugs also saw a big year at FDA in 2015 in terms of approvals. In particular the agency called out four new treatments for multiple myeloma: Johnson & Johnson’s Darzalex (daratumumab), Bristol-Myers Squibb’s Empliciti (elotuzumab), Novartis’ Farydak (panobinostat), and Takeda’s Ninlaro (ixazomib), as well as Roche’s Alecensa (alectinib) and AstraZeneca’s Tagrisso (osimertinib), to treat certain patients with non-small cell lung cancer, Roche’s Cotellic (cobimetinib), to treat certain patients with metastatic melanoma, Taiho’s Lonsurf (trifluridine and tipiracil), for the treatment of certain patients with metastatic colorectal cancer and Yondelis (trabectedin) for the treatment of soft tissue carcinoma.


The approvals came via a number of different regulatory pathways, many of which were expedited.

Fourteen of the approvals (31%) in 2015 came via the fast track designation (potential to address unmet needs), while 10 (22%) were so-called breakthrough therapies (meaning preliminary clinical evidence demonstrating substantial improvement over other therapies), 24 (53%) were priority review drugs (meaning a six month review period instead of the standard 10 months) and six of the drugs (13%) were approved under FDA’s accelerate approval program (meaning early approval for a serious or life-threatening illness with benefit over existing therapies).

Novel Drugs 2015 Summary

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