Boxed Warning for CAR-T Cell Therapy, Dupilumab for Pediatric EOE, Wearable Device for Osteopenia, BSI-082 IND for Solid and Liquid Tumors, VCA-894A for Charcot Marie-Tooth, FDA Roundup

New FDA Approvals

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Boxed Warning for CAR-T Cell Therapy: The FDA mandates a boxed warning on all CAR T-cell therapies due to increased secondary cancer risks, affecting six named treatments. This follows investigations into T-cell malignancies in patients treated with BCMA- or CD19-directed therapies. Manufacturers have 30 days to comply or challenge this requirement, reflecting the FDA's stance that benefits still outweigh risks.

Dupilumab for Pediatric EOE: The FDA has approved dupilumab for children aged 1-11 with eosinophilic esophagitis (EoE), expanding from its prior approval for older patients. Dupilumab, targeting interleukin-4 and -13, treats EoE, an allergic inflammation causing symptoms like heartburn and difficulty swallowing. The approval, based on the EoE KIDS trial, marks the first FDA-approved EoE treatment for this age group.

Wearable Device for Osteopenia: The FDA has cleared Osteoboost, a wearable belt for treating osteopenia, marking a non-drug medical device approach in this field. Developed by Bone Health Technologies, it uses low-intensity vibrations on the spine and hip, inspired by NASA's bone loss prevention methods. Clinical trials show its effectiveness in maintaining bone health, and it was reviewed under the FDA’s De Novo process with Breakthrough Device Designation.

BSI-082 IND for Solid and Liquid Tumors: The FDA approved an IND application for BSI-082, a novel antibody targeting SIRPα and β to enhance tumor-associated macrophages' phagocytic activity. Developed by Biosion USA, Inc., BSI-082 covers over 90% of human populations and avoids broad toxicity typical in CD47-targeting therapies. Its in vivo efficacy, especially when combined with other antibodies, shows significant anti-tumor effects in animal models.

VCA-894A for Charcot Marie-Tooth: The FDA approved an IND application for VCA-894A by Vanda Pharmaceuticals to treat Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S). VCA-894A, an antisense oligonucleotide, targets the IGHMBP2 gene variant responsible for CMT2S. CMT2S, a rare condition with symptoms like muscle weakness and sensory impairment, currently has no available treatments; this approval represents a significant step in addressing this genetic disorder.

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