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By: Patrizia Cavazzoni, M.D., Director, Center for Drug Research and Evaluation

The U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) approved a wide range of therapies in 2023 that will help patients and consumers live better and possibly longer lives. 

Therapies For Many Types of Diseases and Conditions 

In 2023, CDER approved 55 novel drugs, as detailed in our annual New Drug Therapy Approvals report. The active ingredient(s) in a novel drug have not been previously FDA-approved. We also took other important actions, such as expanding the indications or patient populations of some previously approved therapies. In addition, we approved new dosage forms or drug formulations and made some prescription drugs available over-the-counter. 

Our 2023 actions target a wide range of disease areas. For example, we approved treatments for infectious diseases, including COVID-19, respiratory syncytial virus (RSV), and HIV. CDER also approved therapies for neurological conditions, such as amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, and migraine. Moreover, we took approval actions for drugs targeting type 2 diabetes in children, different types of anemia, and chronic weight management, among other heart, blood, kidney, and endocrine disorders. 

In the opioid overdose space, we approved drugs to treat opioid use, misuse, and abuse. Specifically, we approved three opioid overdose reversal drugs as over-the-counter products. We also approved new dosage forms of drugs that reverse opioid overdose and treat opioid use disorder, giving patients more treatment options. 

With respect to lung diseases, our approval actions included therapies for asthma attacks and cystic fibrosis. For gastrointestinal conditions, we approved drugs for inflammatory bowel disease and pediatric functional constipation. 

In oncology, approvals included treatments for colorectal, prostate, lung, and low-grade gliomas (tumors that start in the brain). In the area of women’s health, CDER approved treatments for postpartum depression, hot flashes due to menopause, and over-the-counter oral contraception.

New Drugs for Rare Diseases 

Our commitment to the rare disease community continued in 2023. More than half (28 of 55, or 51%) of the novel drugs approved in 2023 were approved to prevent, diagnose or treat a rare disease or condition, for which they received an orphan-drug designation. 

In addition to ALS, some approvals to prevent, diagnose or treat rare diseases included drugs for Friedreich’s ataxia, an inherited, degenerative disease that damages the nervous system; paroxysmal nocturnal hemoglobinuria, a disease that causes red blood cells to break apart; and activated phosphoinositide 3-kinase delta, a genetic disorder that impairs the immune system. For rare cancers specifically, we approved therapies for mantle cell lymphoma, an aggressive form of non-Hodgkin’s lymphoma, and nasopharyngeal carcinoma, a head and neck cancer.

Efficiencies in Bringing Therapies to Market

CDER recognizes many patients need treatments quickly, and we aim to accomplish this through efficiency in our review process: 

• User Fee Goal Performance: Of the 55 novel drugs approved in 2023, CDER met or exceeded its Prescription Drug User Fee Act goal dates for 49 of these approvals (89%).  A number of delayed reviews in 2023 were due to COVID-19-related foreign travel restrictions, which led to postponement of some inspections during the Public Health Emergency.
• First Cycle Approvals: In 2023, CDER approved 46 of the 55 novel drugs (84%) on the “first cycle.” 
• Approvals Before Other Countries: 35 of the 55 novel drugs approved in 2023 (64%) were approved in the U.S. before being approved in other countries.
• Expedited Programs for Serious Conditions: The FDA’s four broadly applicable programs to facilitate and expedite development and review of drugs for serious or life-threatening conditions include: Fast Track designation, Breakthrough Therapy designation, Priority Review designation, and Accelerated Approval. In 2023, 36 of CDER’s 55 drug approvals (65%) used one or more of these expedited programs.

Biosimilar Approvals 

In 2023, CDER approved five biosimilars, including three biosimilars for reference products that previously did not have a corresponding biosimilar. To date, CDER has approved 45 biosimilars for 14 reference products. Several products were approved as interchangeable biosimilars, which are biosimilars that may be substituted for the reference product at the pharmacy similar to how generics are substituted, subject to state law. To date, CDER has approved seven interchangeable biosimilars.

The 2023 New Drug Therapy Approvals Report 

The 2023 New Drug Therapy Approvals report has more details about novel and other notable drug approvals. Related, the FDA’s Center for Biologics Evaluation and Research (CBER) also approved important therapies. For more information about CBER actions, visit CBER’s web page for 2023 Biological Approvals.  

Deciding whether to approve a drug is a collaborative, well-coordinated process. It often involves expertise from throughout our center and agency, as well as external parties. We take our regulatory decision-making seriously, because we know our drug approval actions affect the health and well-being of patients and consumers nationwide. I want to thank my colleagues for their dedication to reviewing and approving these drugs, which will benefit many people in years to come.