A banner year for new drugs

Last year was a high-water mark for drug approvals, with the U.S. Food & Drug Administration green-lighting 46 new molecular entities. By mid-June, FDA had given its nod to as many new molecules as were approved in all of 2016.

The 2017 number surpassed the nearly 20-year peak reached in 2015, when drug companies pushed through 45 new products.

Cancer treatments represented just over a quarter of all new molecules approved last year, though many work by the same mechanism of action as already-marketed drugs do. Those include two more CDK4/6 inhibitors, two more PD-L1 inhibitors, and more compounds that block the proteins PARP, BTK, and ALK.

Rare diseases also continued to be a focal point for drug developers. Some 41% of new drugs approved in 2017 had “orphan” status, granted to treatments for diseases that affect fewer than 200,000 people.

Although the biotech industry has been energized by the potential for new technologies and therapeutic modalities to tackle difficult-to-treat diseases, small molecules continue to dominate FDA’s docket. Conventional small-molecule drugs represented almost two-thirds of the new molecular entities approved last year.

Still, compared to a decade ago, the new drug list last year featured a wider range of modalities, including enzyme replacement therapies for rare diseases, antibody-drug conjugates, and peptides.

Industry insiders will notice that the tally does not include three drugs from two new therapeutic approaches that garnered many headlines in 2017. Missing are Kymriah and Yescarta, the first two marketed chimeric antigen receptor (CAR) T-cell therapies, and Luxturna, the first gene therapy approved for a genetic condition.

C&EN has long tracked FDA’s actions on new molecular entities, and these cellular treatments—the approval of which the agency called “historic”—fall outside that category. But even without including these groundbreaking therapies in the total, the year was a strong one for the drug industry.