What are "Novel" Drugs?

"Novel" drugs are new drugs never before approved or marketed in the U.S.

The term "novel" drugs refers to medications that are entirely new to the U.S. market, offering unique mechanisms of action or targeting previously untreated conditions. In 2024, the FDA approved 48 novel therapies through its Center for Drug Evaluation and Research (CDER). These include treatments for familial chylomicronemia syndrome, non-small cell lung cancer, and a radiographic contrast agent, among others. This comprehensive list reflects the ongoing advancements in medicine aimed at addressing unmet needs and improving patient outcomes worldwide

The 2024 approvals highlight significant progress in areas such as oncology, cardiology, and rare diseases. Standout examples include Tryngolza (olezarsen) for familial chylomicronemia syndrome and Bizengri (zenocutuzumab-zbco), a dual-action therapy for non-small cell lung cancer and pancreatic adenocarcinoma. Other approvals, like Ziihera (zanidatamab-hrii) for HER2-positive biliary tract cancer and Attruby (acoramidis) for transthyretin amyloidosis, demonstrate the FDA’s focus on innovative solutions for both common and rare diseases.

Beyond treatments, the FDA also advanced diagnostic tools, such as Flyrcado (flurpiridaz F 18), a radioactive diagnostic agent for assessing myocardial ischemia, underscoring the critical role of diagnostics in precision medicine

FDA Novel Drug Therapy Approvals for 2024

The table below is a running list of CDER’s novel drug approvals for 2024.

The FDA’s 2024 novel drug approvals underscore the dynamic evolution of the pharmaceutical landscape, where innovation continues to meet pressing medical needs. From rare genetic disorders to advanced cancer treatments, these therapies not only expand the possibilities of modern medicine but also set the stage for future breakthroughs. As we reflect on this year's progress, it is clear that each approval represents a step forward in enhancing patient care and tackling some of the world’s most challenging health conditions.