The Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research approved 41 new molecular entities in 2014, including several for cancer, type 2 diabetes, hepatitis C, and other serious diseases.
In its “Novel New Drugs 2014 Summary” report, the CDER focused on the quality of several new medications approved, in addition to the quantity. “Many of the 41 new drugs have the potential to add signficant clinical value to the care of thousands of patients with serious or life-threatening diseases,” wrote John Jenkins, MD, director of CDER’s Office of New Drugs, in an FDA Voice blog post on the report.
The approved drugs include nine new medications for cancer, including Lynparza, Blincyto, Keytruda, Zydelig, and Sylvant; four drugs to treat type 2 diabetes, including Farxiga, Tanzeum, Jardiance, and Trulicity; two new drugs for hepatitis C – Viekira Pak and Harvoni; antibiotics to treat serious skin infections including Dalvance, Sivextro (oral and injection), and Orbactiv; and Zontivity for cardiovascular disease.
Notably, 17 (41%) of the new drugs were identified as “First-in-Class,” an indicator of a drug’s degree of innovation. “The total for First-in-Class approvals in 2014 approaches the highest yearly total of 20 reported in 2012,” Jenkins wrote.
In another major advancement, 17 (41%) of the novel new drugs were approved to treat rare or “orphan” diseases affecting 200,000 or fewer Americans. “This is the highest yearly total of such drugs ever – surpassing the previous high of 13 from 2012. These approvals are particularly signficiant because patients with rare diseases often have few or no drugs available to treat their conditions,” Jenkins wrote.
For example, Vimizim was approved to treat mucopolysaccharidosis type IVA, Impavido was approved to treat forms of the rare tropical disease called leishmaniasis, Sylvant was approved to treat multicentric Castleman’s disease, Cerdelga was approved to treat Gaucher disease, Esbriet and Ofev were approved to treat idiopathic pulmonary fibrosis (IPF), and Myalept was approved to treat the complications of lipodystrophy associated with a deficiency of the hormone leptin.