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P-BCMA-101 granted orphan drug designation in relapsed/refractory multiple myeloma

May 14, 2019

The U.S. Food and Drug Administration (FDA) has granted P-BCMA-101 orphan drug designation in relapsed/refractory multiple myeloma (RRMM). P-BCMA-101 was previously granted Regenerative Medicine Advanced Therapy (RMAT) status by the FDA in November 2018. 1,2

About P-BCMA-101: 1-3

  • P-BCMA-101 is an anti-BCMA chimeric antigen receptor (CAR) T-cell therapy
  • The results from the phase I trial ( NCT03288493) were presented during the 60 thAnnual Meeting and Exposition of the American Society of Hematology (ASH) 2
    • N = 25
    • Median lines of prior therapy: 7
    • CAR T construct: human CD3/41BB
    • Dose escalation study of 0.5–5 x 10 8/kg
    • The overall response rate (ORR) was 48%
    • ORR at the optimal dose: 64% (7/11)
    • Cytokine release syndrome grade ≥3: 32%
    • Neurotoxicity grade ≥3: 12%
  • A phase II trial is expected later in 2019
  • Orphan drug designation may be given to drugs that treat diseases that affect <200,000 people in the U.S
  1. FDA Grants Orphan Drug Designation to P-BCMA-101 CAR Therapy for R/R Multiple Myeloma.[accessed 2019 May 14]
  2. FDA Regenerative Medicine Advanced Therapy designation for P-BCMA CAR T-cell therapy.[accessed 2019 May 14]
  3. Gregory T.  et al. Efficacy and Safety of P-Bcma-101 CAR-T Cells in Patients with Relapsed/Refractory (r/r) Multiple Myeloma (MM). Abstract #1012. ASH 60th Annual Meeting and Exposition, San Diego, CA