Product approval information is indicated for adults and children with hemophilia B for: (1) On-demand treatment and control of bleeding episodes, (2) Perioperative management, (3) Routine prophylaxis to reduce the frequency of bleeding episodes.
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Product approval information is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia B (congenital factor IX deficiency or Christmas disease)
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Product approval information is indicated for routine prophylaxis against angioedema attacks in adolescent and adult patients with Hereditary Angioedema (HAE).
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Product approval information is indicated for treatment of acute abdominal or facial attacks of hereditary angioedema (HAE) in adult and adolescent patients.
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CBER 2026 Orphan Approvals (new BLAs)
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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
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Product approval information is indicated for Treatment of infant botulism caused by type A or type B in patients below one year of age.
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For the treatment of adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy. Axicabtagene ciloleucel is not indicated for the treatment of patients with primary central nervous system lymphoma.
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Product approval information is indicated for postexposure prophylaxis in high risk individuals. High risk groups include:
o Immunocompromised children and adults,
o Newborns of mothers with varicella shortly before or after delivery,
o Premature infants,
o Infants less than one year of age,
o Adul
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Is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician(s).
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FDA guidance and regulatory information, specific FDA projects related to monitoring and assessment of MCMs, ongoing FDA work related to medical product assessment in general, and key efforts relevant to MCM monitoring and assessment outside of FDA
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Coronavirus (COVID-19) Update: Daily Roundup
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