FDA Approval of 'Novel' New Drugs Up Sharply in 2014

January 22, 2015

The Center for Drug Evaluation and Research (CDER) at the US Food and Drug Administration (FDA) approved 41 novel new drugs in 2014, 14 more than in 2013 and the highest number in nearly 20 years, the FDA announced last week.

The FDA attributed the high output in part to programs that it created to expedite the review of new drugs and address charges that the agency was a regulatory slowpoke.

As defined by the FDA, novel new drugs are "truly innovative" molecular entities or new therapeutic biologics that have not been previously greenlighted by the agency. In other words, new- dosage versions of drugs already on the market and generic formulations don't count.

The 41 novel new drugs approved in 2014 easily topped the yearly average of 25 since 2005. They included eight drugs for various kinds of cancer, four for type 2 diabetes, two for hepatitis C, and four antibiotics.

Representing an all-time high, 17 of the 41 drugs treat rare diseases that affect 200,000 or fewer Americans. Noteworthy members of this group, the FDA said, include eliglustat (Cerdelga, Genzyme Corporation) for Gaucher disease, oral miltefosine (Impavido, Knight Therapeutics Inc) for a tropical disease called leishmaniasis, and siltuximab (Sylvant, Janssen Pharmaceuticals, Inc) for multicentric Castleman's disease, a blood disorder.

Seventeen of the 41 drugs also were first in class. The FDA singled out three — ledipasvir/sofosbuvir (Harvoni, Gilead Sciences, Inc) for hepatitis C, pembrolizumab (Keytruda, Merck Sharp & Doehme Corp) for unresectable or metastatic melonama, and vorapaxar (Zontivity, Merck Sharpe & Dohme Corp) to reduce the risk for thrombotic cardiovascular events.

The agency cited several measures of how expeditiously it evaluated these drugs:

  • More than three quarters of the 41 novel new drugs were okayed in the first cycle of review, without requests for additional information that would drag out the process.

  • Almost two thirds of the drugs were approved in the United States before they were approved in other countries.

  • Forty of the 41 drugs were approved on or before a deadline date that the agency had set for completing its review.

How the FDA Cuts Red Tape

The CDER uses four regulatory pathways to speed safe and effective drugs to market. All played a significant part in the CDER's approval output last year.

Applications for 61% of the 41 innovative drugs approved in 2014 were marked for priority review. Deeming such drugs as possibly significant advances in medical care, CDER attempts to make its decision within 6 months instead of the standard 10. An example in this category was tedizolid phosphate (Sivextro, Cubist Pharmaceuticals) for skin infections.

Another 41% of the drugs received a fast track designation because they potentially addressed an unmet medical need. Among other things, this route allows CDER to begin reviewing portions of a drug application before it is submitted in its entirety. A drug for ulcerative colitis and Crohn's disease called vedolizumab (Entyvio, Takeda Pharmaceuticals Co, Ltd) was approved this way.

The CDER categorized roughly 1 in 5 of last year's novel new drugs as breakthrough therapies because preliminary clinical evidence suggested substantial improvement over other available medications. In addition to entering a drug such as this in its fast track program, the FDA gives the manufacturer more intensive guidance on efficiently developing it. One such drug was pirfenidone (Esbriet, InterMune, Inc) for idiopathic pulmonary fibrosis.

Yet another 1 in 5 drugs made it into the CDER's accelerated approval program because they treat a serious or life-threatening illness while offering benefits over current therapies. The CDER will approve such a drug on the basis of a surrogate or intermediate clinical endpoint such as a laboratory measure that likely predicts an improvement in how the patient feels or functions. Once on the market, the drug must undergo further studies to confirm the clinical benefit. An example from 2014 was droxidopa (Northera, Lundbeck, Inc) for neurogenic orthostatic hypotension.

More information about the FDA's approval of novel new drugs in 2014 is available on the agency's website.

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