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The numbers of new molecular entities approved in 2019 are close to or exceed FDA’s performance in most previous years.
FDA did not set any records last year in approving new drugs and biologics for market, but it did speed a number of important new therapies to patients, continuing to demonstrate its flexibility and support for biomedical innovation. Based on its criteria, the Center for Drug Evaluation and Research (CDER) approved some 50 new molecular entities (NMEs) in 2019, a count that may vary according to different definitions. In addition, the Center for Biologics Evaluation and Research (CBER) approved the first gene therapy for spinal muscular atrophy, Novartis’ Zolgensma, as well as new vaccines for Ebola (from Merck) and Dengue (Sanofi). While the totals fall short of the record-setting 59 NMEs approved in 2018, the numbers are close to or exceed the agency’s performance in most previous years.
Probably more important than the number is the wide range of breakthrough therapies moving through the agency’s review process, including nearly a dozen cancer treatments, some with innovative modalities. Speedy approvals at the end of the year included the first PARP inhibitor to treat advanced pancreatic cancer, Lynparza, from AstraZeneca and Merck. FDA also approved two antibody-drug conjugates (ADCs), one for urothelial cancer from Seattle Genetics and Astellas Pharma, and another for advanced breast cancer from Daiichi Sankyo and AstraZeneca.
Earlier, Vertex Pharmaceuticals gained a fast approval for a new triple-combo treatment for cystic fibrosis, Trikafta, while new therapies for complicated urinary tract infections came from Shionogi and Merck. Particularly noteworthy is the success of two new drugs for sickle cell disease, which largely affects African Americans. These include Novartis’ Adakveo and Oxbryta from Global Blood Therapeutics. The list also includes important new drugs for postpartum depression from Sage Therapeutics, Intra-Cellular’s Caplyta for schizophrenia in adults, new migraine therapies from Eli Lilly and from Allergan, plus a new Novartis drug for a relapsing form of multiple sclerosis.
Most surprising was FDA’s decision in December to approve Sarepta Therapeutics’ second drug for Duchenne muscular dystrophy, Vyondys 53. The agency had rejected the application only four months earlier.
Speedy review from FDA, as well as high launch prices for many critical new drugs, will continue to raise controversy. Some critics claim the agency is moving too quickly in bringing new therapies to market based on limited testing, while others argue that FDA remains too slow in addressing the critical needs of many patients. And high price tags, particularly for some treatments for larger patient populations such as sickle cell disease, are sure to accelerate calls for pharmaceutical price controls. So far, Congress has stepped back from legislation to limit outlays for prescription drugs, but the debate will be aggravated by a number of new medicines with six-figure annual costs, as well as a large number of annual January increases in list praises for hundreds of brand medicines.