CBER 2025 Orphan Approvals (new BLAs)

Description of CBER’s Rare Disease Program, including a description of the program, selected activities, examples of collaborations internal and external to CBER, and frequently asked questions.

Products that are regulated as Biologics License Applications (BLAs), New Drug Applications (NDAS), Premarket Approvals (PMAs), and 510(k)s.

Use of Artificial Intelligence and Machine Learning (AI/ML) has rapidly become an important component of everyday life. This includes using AI/ML in the development of Medical Products, including Biologics. This page provides an overview of CBER’s activity in AI/ML.

CBER’s Manufacturers Assistance and Technical Training Branch provides assistance and training to industry, including large and small manufacturers and trade associations, and to respond to requests for information regarding CBER policies and procedures.

This is the main page for CBER’s Biologics Effectiveness and Safety (BEST) System.