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CBER 2022 Orphan Approvals (new BLAs)

This list reflects information regarding the applications as of the approval date. It is not updated with regard to applicant or application status changes. The applications are listed by date of approval in reverse chronological order.

Trade Name/Proper Name Indication Manufacturer/License Number Approval Date


Indicated for the prevention of recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older, following antibiotic treatment for recurrent CDI.

Ferring Pharmaceuticals Inc.



Indicated for treatment of adults with Hemophilia B (congenital Factor IX deficiency) who: currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

CSL Behring LLC



Indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).

bluebird bio, Inc.



Indicated for the treatment of adult and pediatric patients with ß-thalassemia who require regular red blood cell (RBC) transfusions.

bluebird bio, Inc.



Is indicated for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

Janssen Biotech, Inc.



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