In 2020, the United States Food and Drug Administration (FDA) not only met all goal dates for approvals by the Center for Drug Evaluation and Research (CDER) but also sped up the approval process for many novel drugs approved throughout the year. For the year, 53 novel drugs were approved (a rough average of one per week!), including first-time approvals and older drugs that were approved for new uses.
Patrizia Cavazzoni, MD, acting director of CDER, noted that all Prescription Drug User Fee Act (PDUFA) goal dates were met for the year’s novel approvals, and all but four of the approvals occurred on the first cycle. The rate of 92% approval on the first cycle is an improvement over the average of 85% first-cycle wins for 2011-2019 novel drug approvals.
“New drug therapies for patients with rare diseases are often among the most important approvals. Patients with rare diseases frequently have few or no drugs available to treat their condition — and for them, approvals of so-called “orphan” drugs can mean new hope for an enhanced quality of life, and in some cases, increased survival,” Cavazzoni said in a post announcing the strong year for new drug approvals.
A wide variety of patients will now be able to receive potentially life-saving drugs for their conditions, including patients who suffer from:
- Various infectious diseases, including COVID-19, HIV-1, chronic hepatitis C, hospital-acquired bacterial pneumonia, ventilator-associated bacterial pneumonia, and flu prevention
- Parkinson’s disease, migraines, and multiple sclerosis
- Heart failure, osteomalacia, and growth hormone deficiency
- Polyarticular juvenile idiopathic arthritis and ulcerative colitis
- Cancer, including twelve notable new treatments for lung cancer, four new notable therapies for various forms of breast cancer, and new therapies for urothelial cancer, colorectal cancer, prostate cancer, and bladder cancer.
Out of the 53 novel drug approvals, 21 of them are considered first-in-class. Of the first-in-class approvals, Rukobia was approved as a new antiretroviral for heavily pretreated individuals with HIV and Koselugo was approved to treat neurofibromatosis type 1.
In 2020, more than half (31 of 53, or 58%), of all novel approvals were for drugs to treat patients with rare diseases. and sponsors of those drugs received incentives under the Orphan Drug Act. Two of the drugs approved in this class included the first oral therapy for spinal muscular atrophy and an oral treatment for hereditary angioedema. Lampit also fell into this category, for its ability to treat Chagas disease, despite the fact that it was developed in the 1960s.
Expedited Reviews
Most of the novel drugs approved last year (36 of the 53) wound through the approvals process through one or more expedited approval pathways.
An impressive 32% of 2020 novel approvals (17 of the 53) were made through the Fast Track designation, which “speeds new drug development and review by increasing the level of communication between FDA and drug developers, and by enabling CDER to review portions of a drug application ahead of the submission of the complete application.”
Breakthrough therapies, which are drugs used for serious of life-threatening diseases for which there is unmet medical need and for which there is preliminary clinical evidence that demonstrates the drug may result in substantial improvement on a clinically significant endpoint over other available therapies. 42% of novel drugs approved in 2020 (22 of 53) received breakthrough therapy status.
Priority review was granted to over half (30 of 53, or 57%) of novel drugs in 2020. The 30 drugs were determined by the agency as potentially being able to “provide a significant advance in medical care.”
Roughly one quarter of the novel drugs (12 of 53) approved in 2020 were approved under the Accelerated Approval program. This program allows more flexibility in determining endpoints for clinical trials and is reserved for drugs offering a benefit over treatments that are currently available to address a condition that is serious or life-threatening.