FDA drug approvals holding steady at 44 YTD in 2024

YTD 2024 FDA approvals: By the numbers

44
Novel Drug Approvals

• As of December 5, 2024, the FDA had approved a total of 44 novel drugs, spanning a diverse array of therapeutic areas and patient populations.
• These new treatments address both widespread public health concerns, such as cardiovascular disease and COPD, and more
  narrowly defined conditions. Those include rare metabolic disorders and targeted oncology indications.

Therapeutic categories

• 9 Oncology treatments (including lung, pancreatic, breast & biliary tract cancers)
• 3 Cardiovascular therapies (e.g., for transthyretin-mediated amyloidosis and severe hypertension)
• 3 Diagnostic/Imaging agents (enhancing precision in identifying myocardial ischemia and various cancers)
• Treatments addressing complex conditions like paroxysmal nocturnal hemoglobinuria, alopecia areata, and Niemann-Pick disease type C
• Advanced therapies for metabolic and inflammatory disorders, including atopic dermatitis, non-alcoholic steatohepatitis (NASH), and primary biliary cholangitis

Peak approval months

• March: 7 approvals (spanning hypertension, NASH, and pulmonary arterial hypertension)
• August: 6 approvals (covering graft-versus-host disease, rare genetic conditions, and hypoparathyroidism)
• Sept/Nov: 5 approvals each (including schizophrenia treatments and novel agents for advanced cancers)

Notable FDA approvals

• Bizengri: A targeted therapy for non-small cell lung and pancreatic cancers, offering new hope for previously limited treatment pathways
• Attruby: Addresses transthyretin-mediated amyloidosis, a rare yet debilitating condition, improving cardiac function and quality of life
• Ziihera: A HER2-positive biliary tract cancer treatment, expanding the range of targeted therapies available for this challenging malignancy
• Exblifep: Combines cefepime and enmetazobactam to tackle complicated urinary tract infections, including drug-resistant strains
• Kisunla: A new Alzheimer’s disease treatment offering measurable cognitive benefit, contributing to a growing field of neurodegenerative therapies.

Source: FDA Novel Drug Approvals 2024 (Accessed on December 16, 2024)

As of December 5, 2024, the FDA had approved 44 new drugs, tracking close to the ten-year rolling average of 46 annual approvals. This pace, however, trails 2023’s 55 approvals.

In oncology, Bizengri (zenocutuzumab-zbco) for NRG1 fusion-positive cancers and Ziihera (zanidatamab) for HER2-positive biliary tract cancer highlighted advances in precision medicine; both drugs tap molecular targeting.

The rare disease space saw progress with the approval of Attruby (acoramidis) for transthyretin-mediated amyloidosis and Augtyro (repotrectinib) for ROS1-positive non-small cell lung cancer.

Novel immunotherapy approaches also gained traction with approvals like Zepbezgo (telisotuzumab vedotin-gxzm) for advanced non-small cell lung cancer.

Patient-centric drug delivery innovations included the approval of Xacduro (sulbactam-durlobactam), which established a new treatment paradigm for drug-resistant infections, and Iomervu (iomeprol), which improved diagnostic capabilities with enhanced radiographic contrast agents. Approximately 20% of approved therapies YTD in 2024 used biomarker-driven patient selection or targeted therapeutic mechanisms.

Precision medicine and targeted therapies

In addition, approvals such as vorasidenib (Voranigo) for IDH1/2-mutant grade 2 gliomas and multiple drugs for non-small cell lung cancer (e.g., Bizengri, Lazcluze) highlight ongoing precision-based strategies. Regulators increasingly considered genetic and molecular markers in approval decisions.

Oncology’s dominant role

As is often the case, oncology approvals remained a central focus. In addition to incremental expansions of existing therapies, 2024 saw first-in-class treatments, including engineered cellular therapies like afamitresgene autoleucel (Tecelra) for synovial cell sarcoma. YTD data show a broadening application of cell and gene therapies beyond hematological malignancies to solid tumors and rarer cancer types. HER2-positive biliary tract cancer (Ziihera) and niche indications such as synovial cell sarcoma benefited from targeted and cellular approaches.

Immunotherapy and administration advances

Immunotherapy approvals, including new subcutaneous formulations (e.g., Tecentriq Hybreza), reflect a drive to streamline treatment logistics and reduce patient burden. The availability of subcutaneous and at-home administration for complex biologics indicates a move toward more accessible care models, without compromising efficacy.

Rare disease therapies

2024 saw multiple treatments approved for conditions with high unmet need, including gene therapies and enzyme replacement strategies for rare genetic disorders. Therapies for Niemann-Pick disease type C (Aqneursa, Miplyffa), primary biliary cholangitis (Ebglyss, Iqirvo), and conditions like WHIM syndrome (Xolremdi) highlight the FDA’s continued support for treatments that address small patient populations.

Drug delivery and formulation advances

Another trend was the conversion of intravenously administered therapies to subcutaneous forms, as well as the use of hyaluronidase-based technologies to improve drug distribution. Approvals for subcutaneous variants and agents with novel delivery systems (e.g., flurpiridaz F 18 for cardiac imaging) show that convenience and accessibility are integral considerations.

Breakthroughs in rare and complex diseases

Many of this year’s approvals focus on conditions that previously had limited or no therapeutic options.

• Treatments for Niemann-Pick disease type C, paroxysmal nocturnal hemoglobinuria (PNH), and pediatric low-grade glioma reflect a push toward precision medicine and improved patient outcomes in smaller populations.
• Additionally, therapies addressing Duchenne muscular dystrophy, WHIM syndrome, and advanced-stage esophageal cancer
  highlight the FDA’s commitment to supporting novel approaches that could significantly change patients’ lives.