By Patrizia Cavazzoni, M.D., Director, Center for Drug Evaluation and Research, FDA
Twitter: @US_FDA
The U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) approved a wide variety of safe and effective new therapies in 2022, even as our nation continues to face the COVID-19 pandemic.
Therapies For a Wide Range of Diseases and Conditions
In 2022, CDER approved 37 new drugs never before approved or marketed in the U.S., known as “novel” drugs, as noted in our annual New Drug Therapy Approvals report. We also approved drugs in new settings, such as for new uses and patient populations.
The 2022 approvals target many different disease areas. For example, we approved treatments for a range of infectious diseases, including COVID-19, HIV, smallpox, influenza, and H. pylori infection (a bacterial infection in the stomach). Also approved were therapies for two severe and progressive neurological conditions, amyotrophic lateral sclerosis and spinal muscular atrophy.
CDER approved drugs for a range of heart, blood, kidney, and endocrine disorders, including drugs for type 1 and 2 diabetes, anemia, kidney impairment, and chronic weight management. We also approved therapies for autoimmune, inflammatory, and lung diseases, such as inflammatory bowel disease, nutritional deficiencies, lupus nephritis, arthritis, eosinophilic esophagitis (a chronic inflammatory disorder), and psoriasis.
In oncology, approvals included treatments for lung cancer, prostate cancer, uveal melanoma (a rare cancer that develops in a part of the eye called the uvea), and types of breast cancer, among other kinds of cancers.
New Drugs for Rare Diseases
Our commitment to the rare disease community continued in 2022 with more than half (20 of 37, or 54%) of our novel drug approvals for patients with rare diseases, who often have no or few treatment options.
For example, we approved a therapy for acid sphingomyelinase deficiency (Niemann-Pick disease type A, B, A/B), an inherited disease that affects fat metabolism; generalized pustular psoriasis, a life-threatening skin disease; and obstructive hypertrophic cardiomyopathy, a disease in which the heart muscle thickens. We also approved a treatment for a form of impaired kidney function.
Drug Review Efficiencies
CDER recognizes many patients need treatments quickly, and we emphasize both thorough assessment and efficiency in our review process:
- User Fee Goal Performance: In 2022, CDER met or exceeded its Prescription Drug User Fee Act goal dates for 36 of the 37 novel drugs approved (97%).
- First Cycle Approvals: In 2022, CDER approved 28 of the 37 novel approvals (76%) on the first cycle. This differs from CDER not approving the drug on the first cycle and possibly asking the applicant for more information.
- Approvals Before Other Countries: 25 of the 37 novel drugs approved in 2022 (68%) were approved in the U.S. before being approved in other countries.
- Expedited Programs for Serious Conditions: the FDA has four programs to facilitate and expedite development and review of drugs for serious or life-threatening conditions: Fast Track designation, Breakthrough Therapy designation, Priority Review designation, and Accelerated Approval. In 2022, 24 of CDER’s 37 drug approvals (65%) used one or more of these expedited programs.
Biosimilar Approvals
In 2022, CDER approved the 40th biosimilar, a significant achievement in biosimilar product development. CDER also approved two interchangeable biosimilars, which are biological products that may (subject to state law) be substituted without the intervention of a prescriber, similar to how generic drugs are substituted for brand name drugs. There are now four approved interchangeable biosimilars.
The 2022 New Drug Therapy Approvals Report
The number of drug applications CDER received in 2022 was similar to the past few years (and higher than historical norms), demonstrating that innovation remains strong in the drug development sector. CDER only approves drug and biologic applications that meet our high regulatory standards.
The New Drug Therapy Approvals report mentioned above has more details about these drug approvals, including a narrative about the notable approvals. The FDA’s Center for Biologics Evaluation and Research (CBER) approves important therapies as well. For more information about CBER actions, visit CBER’s web page for 2022 Biological Approvals.
The decision to approve a therapy involves a multidisciplinary team of experts from across CDER who carefully review drug applications and make benefit-risk calculations. I am grateful to my CDER colleagues for their expertise, dedication, and collaboration in the drug review process. The drugs we approved in 2022 will improve the lives of patients and consumers, and I am looking forward to another productive year in 2023.
This article was originally published on FDA Voices and is reprinted here with permission.
