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CBER 2023 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
CASGEVY Indicated for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises.  Vertex Pharmaceuticals Inc 12/8/2023
LYFGENIA Indicated for the treatment of sickle cell disease (SCD) in patients 12 years and older with a history of vaso-occlusive events. bluebird bio Inc 12/8/2023
ADZYNMA Indicated for prophylactic or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP) Takeda, Pharmaceuticals, U.S.A., Inc 11/9/2023
BALFAXAR For the urgent reversal of acquired coagulation factor deficiency induced by Vitamin K antagonist (VKA, e.g., warfarin) therapy in adult patients with need for an urgent surgery/invasive procedure. Octapharma Pharmazeutika Produktiosges.m.b.H. 07/21/2023
CYFENDUS For post-exposure prophylaxis of disease following suspected or confirmed exposure to Bacillus anthracis in persons 18 through 65 years of age when administered in conjunction with recommended antibacterial drugs. Emergent Product Development Gaithersburg Inc 07/20/2023
ROCTAVIAN Valoctocogene roxaparvovec-rvox is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity <1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test. BioMarin Pharmaceutical Inc. 06/29/2023
LANTIDRA Indicated for the treatment of adults with Type 1 diabetes who are unable to approach target HbA1c because of current repeated episodes of severe hypoglycemia despite intensive diabetes management and education. CellTrans Inc. 06/28/2023
ELEVIDYS Indicated for treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. Sarepta Therapeutics, Inc. 06/22/2023
VYJUVEK Indicated for treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. Krystal Biotech, Inc. 05/19/2023
VOWST To prevent the recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older following antibacterial treatment for recurrent CDI (rCDI). Seres Therapeutics, Inc. 04/26/2023
OMISIRGE Indicated for use in adults and pediatric patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection. Gamida Cell Ltd. 04/17/2023
ALTUVIIIO Indicated for use in adults and children with Hemophilia A (congenital Factor VIII deficiency) for: (1) Routine prophylaxis to reduce the frequency of bleeding episodes; (2) On-demand treatment and control of bleeding episodes; and (3) Perioperative management of bleeding. Bioverativ Therapeutics, Inc. 02/22/2023


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