The U.S. Food and Drug Administration (FDA) approved a total of 60 new drugs in 2021. A new report from the FDA catalogs 50 new approvals by the Center for Drug Evaluation and Research (CDER). Separately, the Center for Biologics Evaluation and Research (CBER) approved 10. These novel medicines offer to transform many debilitating diseases, resulting in improved survival, better health outcomes, enhanced quality of life — and in many cases, they provide a first-time treatment option for patients.
According to the CDER report, 54% (27) of the novel medicines approved last year were first-in-class, providing new options of treating diseases — such as a new treatment for the most common type of lupus as well as another medicine for a serious complication of lupus affecting the kidneys: lupus nephritis. New first-in-class treatments also include new medicines for lung cancer as well as new medicines to treat a common and deadly virus in post-transplant patients. Additional novel approvals last year include medicines to reduce risk of cardiovascular death and heart failure hospitalization, to treat a genetic form of endometrial cancer, to address episodic migraines and the first complete HIV treatment that can be administered once a month.
While the number of medicines approved by the FDA ebbs and flows annually, the significant number of approvals occurring during the pandemic is a testament to the Agency's commitment to the public health and the dedication of biopharmaceutical researchers to drive medical advancements despite the ongoing challenges of COVID-19.
At CBER, five new approved vaccines, including the first FDA-approved COVID-19 occurred in 2021, as well as two new CAR-T cell therapies for certain types of multiple myeloma and lymphoma. CAR-T cell therapies represent a game-changing approach involving permanently altering a patient’s T-cells to recognize, target and kill cancer cells.
Key takeaways from the new CDER report include:
The FDA User Fee Program is Working
The PDUFA program helped speed access to therapies for patients with severe asthma and lupus, among other diseases. Additionally, 38 out of 50 (76%) of the newly approved medicines were approved in the United States before any other country.
Among the novel medicines approved this past year, 37 of them (74%) used one or more expedited programs, and 14 (28%) of them were put through the accelerated approval pathway. Accelerated approval allows for earlier approval, based on a surrogate or intermediate clinical endpoint, of drugs that treat serious conditions and that fill an unmet medical need, all while preserving FDA’s high standards for safety. The pathway has been critical to allowing earlier access to important treatment advances for patients across diseases, especially cancer.
These examples illustrate PDUFA’s effectiveness in helping FDA to fulfill its central mission — to protect and advance the public health — by allowing the Agency to stay abreast of ever-evolving science and have the resources to keep pace with the innovative drugs and biologics entering the review pipeline.
New Uses of Approved Medicines and Expanded Medicine Approval for Pediatric Populations
Continual research and development, even after a medicine has already been approved by the FDA, often results in new uses for a medication, including for treatment earlier in a disease, for different patient populations such as a pediatric population, or for entirely different diseases or conditions. Medical advances that happen after initial FDA approval have resulted in improved survival rates, better quality of life and facilitated improved ease of use leading to enhanced patient outcomes. In 2021 alone, several medicines were approved for uses beyond their original FDA approval including treatments for many different types of cancers, as well as many rare and chronic diseases.
Inclusion of pediatric information in the labeling for many medicines often require additional pediatric-specific studies and the submission of new data to support the safe and effective use of the medicine in pediatric patients. These studies and expanded labeling are critical to providing new treatment options for pediatric populations. For example, a medicine that was originally approved in 2010 for adults with a certain type of irregular heartbeat was approved in 2021 for use to prevent and treat blood clots in patients as young as 8 years old — providing the first oral blood thinning medication for children. Additional approvals in 2021 specifically for pediatric populations include medicines to treat children with sickle cell disease, HIV, hepatitis C, cystic fibrosis, high risk cardiovascular conditions and many more.
Continued Progress for Rare Diseases
Of CDER’s approvals last year, 26 of the 50 medicines (52%) were approved to treat rare or “orphan” diseases. Rare or “orphan” diseases are diseases that affect less than 200,000 people in the U.S. These include treatments for many devastating and life-threatening genetic disorders as well as many rare forms of cancer — including for example:
- A medicine to treat patients one year and older with late-onset Pompe disease, a disease that causes muscle weakness and premature death from respiratory or heart failure.
- A medicine to reduce risk of death due to molybdenum cofactor deficiency type A, which presents in the first few days of life, causing seizures, brain injury and death.
- Medicines to treat rare blood disorders and diseases leading to liver dysfunction and failure.
- Medicine to treat certain types of lung cancer, leukemia and lymphoma.
As we look to the future, biopharmaceutical companies are committed to continuing to research and develop treatments that improve the lives of patients. Furthermore, new developments in medical and fundamental science — including immunotherapies and cell and gene therapies — hold the promise of treating debilitating diseases such as cancer, diabetes and many rare disorders. Fulfilling this promise depends on continuing to modernize the drug development and regulatory framework that PDUFA helps facilitate. In turn, this allows FDA to better serve patients by providing timely, science-based regulatory decisions. It is critical that Congress reauthorizes the PDUFA program before its expiration in September 2022.
To learn more about last year’s advancements from the CDER New Drug Therapy Approvals 2021 report click here.