A Review of Recent Drug Approvals

The approval of new drugs by the FDA provides prescribers and patients with additional therapies and represents advances in treating various diseases, especially when approvals of drugs with novel mechanisms of action provide first-time therapies or expand therapeutic options for disease management.

FDA’s Center for Drug Evaluation and Research (CDER), released in 2023, indicated that 55 novel drugs were approved to treat an extensive array of diseases and conditions.^1,2 According to the CDER, drugs were approved for the treatment of various infectious diseases, including HIV, COVID-19, and respiratory syncytial virus (RSV). Other drugs that were approved in 2023 are indicated for the treatment of asthma, cystic fibrosis, inflammatory bowel disease, opioid use, misuse and abuse, and opioid overdose.^1,2 Other conditions include ALS, Alzheimer’s disease, migraine, type 2 diabetes in pediatric patients, postpartum depression, vasomotor symptoms due to menopause, and oncology drugs for colorectal, prostate, lung, and low-grade gliomas.^1,2 The FDA also notes, “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.”¹ Rett syndrome, paroxysmal nocturnal hemoglobinuria, and activated phosphoinositide 3-kinase delta are rare disease that have gained recent FDA-approved drug therapies. The CDER report also indicates that the CDER approved five biosimilars, including three biosimilars for reference products that did not have a comparable biosimilar. To date, the CDER has approved 45 biosimilars for 14 reference products. The CDER states, “Multiple biosimilars for an approved reference product can enhance competition, which may lead to reduced costs for both patients and our health care system.”¹

The CDER report also notes that the review process for a drug application warrants a collaborative process that requires meticulous and well-organized scientific, regulatory, and policy experts from their office and sometimes other parts of the agency. The CDER wrote, “For each application, we perform a very careful and diligent analysis of safety and effectiveness data, including a benefit-risk analysis that factors in the severity of the disease or condition, the currently available treatment options, and the intended patient population. If the therapy meets the standard for approval, we must reach agreement on the indication, labeling, safety issues, and other considerations.¹

Examples of novel first-in-class approvals in 2023 include:

The CDER also approved drugs for expanded indications for use in pediatric patients, approved some drugs for new formulations of some approved drugs, and also changed and switched some drugs from Rx status to OTC status. For example, in 2023, the CDER expanded the indication of use of Synjardy (empagliflozin and metformin hydrochloride), which was approved originally in 2015 and extended the patient population to include pediatric patients ten years and older in 2023. Also, in 2023, the CDER approved Narcan as a nonprescription drug.

Drugs Approvals for 2024

So far, in 2024, Zelsuvmi (brazier) was approved on January 5, 2024, and is indicated for treating molluscum contagiosum in adults and pediatric patients one year and older. On February 9, 2024, the FDA approved Eohilia (budesonide oral suspension) as a mucoadhesive formulation of the approved corticosteroid budesonide for treating eosinophilic esophagitis.^3,4

Conclusion

As the aging population continues to expand and the number of individuals diagnosed with various diseases and conditions continues to grow, the availability of numerous therapies, including novel therapies, provide patients with valuable therapies that have the potential to improve clinical outcomes and overall health-related quality of life. Pharmacists can be instrumental in providing prescribers and patients with efficacy and safety information about recently approved medications, especially for conditions with first-time therapies. Pharmacists can also be advocates for patients and assist them in working with prescribers and insurance companies to expand access to novel therapies. They can also provide information about manufacturer-sponsored copay assistance programs for eligible patients and prior authorization if needed.

Helpful Resources

• CDER New Drug Approvals Report
• 2023 FDA Drug Approvals

References

1. New drug therapy approvals 2023. CDER. Published January 2024. Accessed February 15, 2024. https://www.fda.gov/media/175253/download?attachment
2. Novel drug approvals for 2023. FDA. Published January 16, 2024. Accessed February 15, 2024. https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2023
3. Novel drug approvals for 2024. FDA. Published January 5, 2024. Accessed February 14, 2024. https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2024
4. New drug approvals. Drugs.com. Published 2024. Accessed February 14, 2024. https://www.drugs.com/newdrugs.html