FDA and CDER: New Drug Therapy Approvals 2023

As we near the end of the year, I often take moments for reflection of the previous year’s successes from myself, family, friends, colleagues, etc. It is important to celebrate successes as we look forward to opportunities in the new year. Within healthcare, the Center for Drug Evaluation and Research (CDER) does the same.

The topic for today discusses novel drug approvals that occurred within the 2023 calendar year. I chose this topic because I enjoy reading about the science of new approvals for any indication and thinking about a medication’s potential for its intended patient population. An approval affects many healthcare environments including community, ambulatory, inpatient, and the pharmaceutical industry. I urge readers to reflect on their own experiences in healthcare and analyze how these approvals will affect healthcare moving forward.

CDER regulates over-the-counter and prescription drugs, including biological therapeutics and generic drugs, and aims to ensure that safe and effective medications are available to patients in the United States. Since 2011, CDER has released an annual report providing an overview of drug approvals from the previous year. The reports are titled, “Advancing Health Through Innovation - New Drug Therapy Approvals [Insert Year].” Years 2021 to the running list of 2024 can be found here. The finalized publishing of the 2024 list will be released in January 2025. Years dating back to 2015 are archived in the FDA database here. The archival of this web page means that the page is no longer updated by the FDA. Please click here to view the 2023 publishing: Advancing Health Through Innovation - New Drug Therapy Approvals 2023.

Each report contains an overview of novel products approved in a respective calendar year. CDER defines novel products as medications that are approved as new molecular entities (NMEs) under New Drug Applications (NDAs) or as Biologic Licensing Applications (BLAs). In other words, a novel product is a therapy that has not previously been approved in the U.S. An NME is utilized to bring a product with an active ingredient that has not been previously approved by the FDA. An NDA is utilized for bringing traditional small-molecule products to market. A BLA is utilized to bring a biologic, a large molecule product derived from living organisms, to market.

Collectively, the 55 approvals cover a multitude of therapeutic areas and indications. One interesting takeaway from the 2023 report is that CDER completed 55 novel drug approvals. Figure 1 below shows that 2023 contains the second-highest number of novel drug approvals in the last 10 years. Of the 55 approvals, 38 are BLAs and 17 are NDAs. Compared to 2022, 2023 saw 50% more novel drug approvals.

The full list of approvals and their indicated therapeutic area are included in Figure 2.

Highlights of the approvals are included in the following list:

• Entyvio (vedolizumab) injection to treat moderate-to-severe ulcerative colitis. The injection allows patients to self-administer the medication after training and avoids the need to travel to an infusion center.
• Leqembi (lecanemab-irmb) injection to treat Alzheimer’s disease.
• Narcan (nifurtimox) over-the-counter medication to reverse the effects of opioid overdose.
• Xacduro (sulbactam and durlobactam) injection to treat hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia caused by Acinetobacter baumannii-calcoaceticus complex.
• Zavzpret (zavegepant) nasal spray to treat acute migraine with or without aura.
• Zurzuvae (zuranolone) tablets as the first oral medication to treat postpartum depression.

Of the 55 approvals, 20 are first-in-class and are included in the following list:

• Daybue, Defencath, Fabhalta, Filspari, Filsuvez, Jesduvroq, Joenja, Lamzede, Miebo, Ogsiveo, Paxlovid, Qalsody, Rivfloza, Skyclarys, Sohonos, Talvey, Truqap, Veopoz, Veozah, Xdemvy
  • Highlights of this list include:
    • Jesduvroq (daprodustat) tablets as the first oral treatment for anemia caused by chronic kidney disease for adults receiving dialysis for at least four months.
    • Veozah (fezolinetant) tablets to treat moderate to severe vasomotor symptoms, or hot flashes, due to menopause.
    • Skyclarys (omaveloxolone) capsules as the first treatment for Friedreich’s ataxia.

I am certainly appreciative of these 55 approvals covering many therapeutic areas including rare diseases. Collectively, the 2023 approvals target various diseases and conditions as well as provide potential therapy for patients who have had few or no treatment options in the past. This shows that medicine continues evolving and developing each year with the opportunity to prolong and/or enhance the lives of patients with one or more diseases or conditions.

A personal takeaway I am appreciative of is the opportunity to better combat opioid overdoses. In 2023, CDER and the FDA approved three opioid overdose reversal medications that are available without a prescription. Eliminating the need for a prescription increases access to these products that can be life-saving therapies. The three medications are Narcan (naloxone) as a nasal spray, Opvee (nalmefene) as a nasal spray, and RiVive (naloxone) as a nasal spray. The difference between Narcan and RiVive is the dosing. Narcan is dosed as 4mg/spray and RiVive is dosed as 3mg/spray. The approval of RiVive for nonprescription use was supported by data showing that similar levels of RiVive reach the bloodstream as an approved prescription naloxone product. FDA Commissioner Robert Califf has stated that the FDA has prioritized access to naloxone products, and the agency welcomes manufacturers of other naloxone products to discuss potential nonprescription programs with the FDA. In other words, manufacturers may develop products with naloxone as the active ingredient and, provided that the data shows similar safety and efficacy as approved products, can receive approval for its developed product. This is what we see with the approval of RiVive, and it is an effort to increase access of opioid overdose reversal products.

I chose the above highlights from the overall approval list as well as the first-in-class approval list because these products are indicated for diseases and conditions that can affect a large percentage of the American population. At the same time, we have spent dedicated time in our respective healthcare classes learning about each of the indications listed above. It is my hope that the publishing of this article results in readers learning about various products and therefore allowing them to discuss these medications with family or friends who may benefit from any of the products in the approval list. In addition, my hope is that readers are better equipped to stay up to date with novel drug approvals and look forward to the release of the Advancing Health Through Innovation 2024 as well as approvals in future years.

*Information presented on RxTeach does not represent the opinion of any specific company, organization, or team other than the authors themselves. No patient-provider relationship is created.