Much has been said about the precipitous drop in FDA approvals during 2016, and in fact retiring FDA new drugs director John Jenkins reported in December that as of Dec. 9, 2016, the U.S. agency had approved just 19 NDAs and BLAs, the lowest number since 2007 (Fig. 1). By year-end, that number had increased slightly, for a total of 22 new drug approvals.
Fig. 1. CDER NME NDAs/BLAs from 1993-2016. Data current as of Dec. 9, 2016. Source: U.S. Food and Drug Administration (http://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProd…).
Jenkins provided several possible explanations for the decrease, including last-minute 2015 approvals of five drugs with 2016 PDUFA dates; fewer NDA filings during 2016; and an increase in complete response letters requiring further action by filers. Further scrutiny of the reasons for this drop will surely keep analysts busy well into 2017; nonetheless, with new launch activity consistently lower on a global scale last year, the U.S. maintained the lead as the most active market (Fig. 2).
Fig. 2. Distribution of new launches by country, 2016.
According to analysis in “The Year’s New Drugs & Biologics, 2016,” from Clarivate Analytics, 44 new drugs and biologics were launched in 2016, nearly 10% fewer than the previous year. In addition, 23 significant new line extensions (i.e., new formulations, new combinations and new indications for previously marketed drugs) were introduced.
Immunomodulators and agents for immunization were the most active therapeutic group (Table I), with nine new products launched. Many of these were new antiviral vaccines, including several new quadrivalent influenza vaccines. Under the heading of anti-infective agents, new drugs and drug combinations for hepatitis C continue to emerge from the pipeline at a high rate, with the first global launches of two new direct-acting antiviral drug combinations, one single anti-HCV agent and a novel interferon. In contrast, there were significantly fewer new cancer drugs introduced as compared to previous years: just four in 2016, versus 14 in 2015, 10 in 2014 and 12 in 2013.
*Does not include line extensions.
**Includes one drug launched for two indications in different therapeutic categories.
In spite of the disappointingly low numbers overall, it should be noted that several significant new products were launched last year. Notable among these were Dengvaxia, the world’s first vaccine against dengue, an infectious tropical disease that causes between 50 million and 100 million cases of symptomatic disease each year; pimavanserin (Nuplazid), the first-ever treatment for psychosis in patients with Parkinson’s disease; the antiepileptic drug Spritam (levetiracetam), the first drug produced using 3D printing technology; and vonicog alfa (Vonvendi), the first and only recombinant treatment for adults with a bleeding disorder known as von Willebrand disease. Seven of the new drugs were first-in-class agents—meaning the first drug with a novel mechanism of action to be approved and launched anywhere in the world—including the exon-skipping agent eteplirsen (Exondys 51) for Duchenne muscular dystrophy, the LFA-1/ICAM-1 interaction inhibitor lifitegrast (Xiidra) for dry eye, and the SMN2 expression enhancer nusinersen (Spinraza) for spinal muscular atrophy. Two of the four new cancer drugs launched were also first-in-class agents: the anti-CD140a (PDGFR-α) MAb olaratumab (Lartruvo) and the B-cell lymphoma-2 (BCL-2) inhibitor venetoclax (Venclexta), indicated for soft tissue sarcoma and chronic lymphocytic leukemia, respectively.
Orphan drugs continue to be a focus of new drug R&D, and 14 of last year’s new launches (including line extensions) had orphan drug status, some for more than one indication. Orphan drugs are also fingered as a driver of increasing drug prices, a topic discussed in Part II of the review.
“The Year’s New Drugs & Biologics” is an annual two-part feature in Drugs of Today, a journal published by Clarivate Analytics. Written by Ann Graul, Patricia Pina, Elisabet Cruces and Mark Stringer, Part I provides a comprehensive look at the previous year’s new approvals and launches, while Part II (written by A. Graul, E. Cruces, Coia Dulsat and Michelle Tracy) offers insight into other important news and issues affecting the pharmaceutical industry.