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CBER Rare Disease Program 

Rare diseases and conditions as defined by the Orphan Drug Act (1983) affect less than 200,000 people in the United States. Most of these disease and conditions currently have no FDA-approved treatment. However, with recent scientific developments and FDA’s commitment to help foster the development of new and innovative medical products to treat rare diseases that were previously considered incurable, there is hope that someday FDA-approved therapies will be available to treat the many rare diseases that currently have no cure.

The Center for Biologics Evaluation and Research (CBER) has a longstanding history of regulating and advancing development of biological products for use in rare diseases and conditions. In addition to regulatory oversight of clinical studies, CBER provides proactive scientific and regulatory advice to medical researchers and manufacturers of complex biological products throughout the development process. In the past, most biological products regulated by CBER and intended or approved for use in rare diseases were derived from plasma and used to treat and manage rare blood disorders, immune deficiencies, and various toxicities. Today, CBER continues to regulate and advance the development of these biologics for rare diseases, as well as other new and innovative biological products, such as cell and gene therapy products, all for an ever-expanding array of rare disease indications.


Learn More About CBER’s Rare Disease Program

CBER Rare Disease Program Highlights

CBER Orphan Approvals

CBER's new Biologics License Application Approvals for novel biologics with orphan drug designation are provided below by year of approval starting with calendar year 2022.

Collaboration Across FDA and Beyond

Collaboration is a hallmark of the CBER Rare Disease Program. CBER staff collaborate with rare disease partners across FDA, such as in developing and providing rare disease-related training for review staff; developing and participating in outreach activities; and developing policy such as rare disease related guidance. CBER also collaborates with external stakeholders and engages in public-private partnerships and consortia on efforts that help advance development of medical products for rare diseases.

Contact Information

For inquiries, please contact: ocod@fda.hhs.gov.

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