2014 FDA Drug Approvals at 18-year High, but How Much New Ground Are We Breaking?

As the New Year bells chimed in 2015, the curtains drew shut on this century’s best year for novel-drug approvals. The U.S. FDA green-lighted 41 new agents for the U.S. market, in fields ranging from orphan diseases (15) to oncology (10) and infectious diseases (13). As is typical, none of the 41 drugs have pronounceable names and we can now look forward to sounding out Ofev (nintedanib) for idiopathic pulmonary fibrosis.

Nomenclature aside, this is seriously good news – foremost for the patient populations that these new medicines will treat. It’s a humbling reminder of the struggles people encounter daily, battling conditions many of us have never heard of. Vanda Pharmaceutical’s Hetlioz, for example, became the first medication approved for non-24-hour sleep-wake disorder, a condition affecting the totally blind. Unable to perceive light, the body is literally in the dark when distinguishing day from night, causing a permanent feeling of jet lag that Hetlioz can now relieve.

First in class means there are others to follow. Perhaps the biggest hype amongst this year’s approvals was the first of the PD-1 inhibitors, Keytruda and Opdivo. Both fall under the banner of cancer immunotherapies – therapies that treat cancer by unleashing the patient’s own immune system. T-cells and B-cells are natural cancer-killing machines, but the immune system has programmed-in safe guards to prevent them from damaging healthy tissue. Many tumor cells exploit this feature by binding PD-1 receptors and down regulating T and B-cell action. The newly approved monoclonal antibodies block PD-1 receptor signaling, exposing the bad-boy cancer cells for the immune system to destroy.

The continued success of monoclonal antibodies gave rise to seven of these new biologics in 2014, all ending with the suffix ‘mab’. There is some method to the naming madness – for the generic terms at least. Monoclonal antibodies are submitted as ‘mab’, while ‘zumab’ denotes a humanized version. Other common drug flavors include ‘ib’, which indicates a small molecule (versus a biologic). An example of these is the tyrosine-kinase class of inhibitors, ‘tinibs’, which work by shutting down that disease-causing pathway. All ‘virs’ are anti-viral medications, as with Viekira Pak approved last year for hepatitis C infection. That combination medicine from Abbvie includes the antivirals ombitasvir, paritaprevir, riotnarvir and dasabuvir and will set you back a cool $83,320. But, then again, nothing on this new approvals list is cheap.

The biologics versus small molecules story also plays out at an industry level. Over the last few decades, biotechnology companies have begun to overtake the pharmaceutical industry’s monopoly on drug development and sales. Despite this, 2014 was a good year for pharma, who have been furiously acquiring exogenous programs of innovation. UK-based AstraZeneca cleared four new entities alone, while simultaneously fighting off an acquisition attempt by big fish Pfizer. Another pharma company, Eli Lilly, scored three new approvals in 2014, no doubt a welcomed relief after a string of phase III calamities.

In all, 2014 was a great year for building up the medical armamentarium, but is it an anomaly? While 2013 wasn’t a golden year; the industry is generally doing well after a dip in the mid 2000’s. As evidence of a trend, Chemical & Engineering News ran a remarkably similar headline two years ago, “New Drug Approvals Hit 16-year High in 2012”. The tally then was 39, up from 30 in 2011.

While the 1996 all-time-high can be partly attributed to an FDA bottleneck that was released by legislative change, the new entities approved in 2014 represent a strong pool of late-stage medicines. Does that conflict with claims the drug development pipeline is in a desperate drought? Yes and no. There is a lack of early- to mid-phase candidates, but the complex design of the drugs seems to be executing strong pivotal trials. With initiatives from the FDA, such as the breakthrough therapy designation, things look promising for future years. The drug development pipeline may seem lacking, but what matters is how many cross the finish line to deliver benefits to future patients.