Did the EMA and FDA Accelerated Approval Pathways Reverse the Drought in Novel Anti-Infectives?

OBJECTIVES: The FDA Safety and Innovation Act (FDASIA, 2012) established fast-track and breakthrough therapy designations for new anti-infectives intended to treat serious or life-threatening infections. FDASIA also introduced the qualifying infectious disease product (QIDP) designation to expedite the review of drugs targeting resistant pathogens. Similarly, the EMA Accelerated Assessment (2005) and Priority Medicines (PRIME, 2016) Scheme expedited the review and approval of medicines of major public health interest or therapeutic innovation and medicines addressing unmet medical needs, respectively. This study assessed the effectiveness of these regulations in bringing novel systemic antibiotics to the market.

METHODS: Observational study including new systemic antibiotics approved by the FDA and the EMA through June 2024. Data were obtained from the FDA and EMA websites. Data collection followed a predetermined protocol using a standardized extraction form. Descriptive statistics were conducted.

RESULTS: Since the enactment of FDASIA, the FDA approved 17 new systemic antibiotics. All approvals were based on non-inferiority trials in patients with infections susceptible to existing treatments. These antibiotics were approved for 30 indications across six different infections, with 16 receiving QIDP designation. Thirteen of these 16 QIDP antibiotics were already under clinical development before FDASIA. Only one infection had labeled indications for drug-resistant pathogens. The EMA approved 12 and rejected 1 of those 17 antibiotics, sponsor companies withdrew applications for two antibiotics, and two were not submitted to the EMA. None of the 12 antibiotics approved by the EMA qualified for accelerated assessment or PRIME development schemes.

CONCLUSIONS: EMA and FDA regulatory initiatives have accelerated the regulatory approval review of antibiotics intended for serious infections caused by resistant or potentially resistant bacteria and aimed to address unmet medical needs. However, there remains a critical need for innovative therapies targeting both susceptible and resistant pathogens to improve patient outcomes, enhance survival, and reduce symptom burden.