Q3 2024
Newsletters and Drug Reports

Newly Approved Aucatzyl and Kebilidi

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On November 8, 2024, the US Food and Drug Administration (FDA) approved  Aucatzyl® (obecabtagene autoleucel; obe-cel), a chimeric antigen receptor (CAR) T-cell therapy for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). Aucatzyl is the third US FDA-approved CAR-T for the treatment of ALL, but is the first CD19 targeted CAR-T. CD19 targeted therapy potentially minimizing side effects using a customized tumor-burden guide which is based on the patient’s bone marrow biopsy take prior to lymphodepletion therapy. The wholesale acquisition cost is $525,000.  The administration cost of Aucatzyl®  can vary but the average is $300K-$500K.

 

On November 13, 2024, the FDA approved Kebilidi™  (eladocagene exuparvovec-tneq) gene therapy for the treatment of children and adult patients with aromatic L–amino acid decarboxylase (AADC) deficiency, including the full spectrum of disease severity. AADC deficiency is a rare autosomal recessive neurometabolic disorder that affects the brain. The genetic mutation leads to a decrease in the amount of neurotransmitters. AADC deficiency is a rare, with an estimated prevalence of between 1:64,000 and 1:90,000 births in the USA. Fewer than 1,000 people in the US have the condition.  The cost of Kebilidi™  has not yet been released by the manufacturer.  The therapy has been approved in the UK since 2022 with a current cost of $3.7 million.