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

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