All content on this site is intended for healthcare professionals only. By acknowledging this message and accessing the information on this website you are confirming that you are a Healthcare Professional. If you are a patient or carer, please visit the International Myeloma Foundation or HealthTree for Multiple Myeloma.

The Multiple Myeloma Hub uses cookies on this website. They help us give you the best online experience. By continuing to use our website without changing your cookie settings, you agree to our use of cookies in accordance with our updated Cookie Policy

Introducing

Now you can personalise
your Multiple Myeloma Hub experience!

Bookmark content to read later

Select your specific areas of interest

View content recommended for you

Find out more
  TRANSLATE

The Multiple Myeloma Hub website uses a third-party service provided by Google that dynamically translates web content. Translations are machine generated, so may not be an exact or complete translation, and the Multiple Myeloma Hub cannot guarantee the accuracy of translated content. The Multiple Myeloma Hub and its employees will not be liable for any direct, indirect, or consequential damages (even if foreseeable) resulting from use of the Google Translate feature. For further support with Google Translate, visit Google Translate Help.

Steering CommitteeAbout UsNewsletterContact
LOADING
You're logged in! Click here any time to manage your account or log out.
LOADING
You're logged in! Click here any time to manage your account or log out.
2019-05-14T11:24:19.000Z

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

May 14, 2019
Share:

Bookmark this article

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 60th Annual 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 108/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. https://www.targetedonc.com/news/fda-grants-orphan-drug-designation-to-pbcma101-car-therapy-for-rr-multiple-myeloma [accessed 2019 May 14]
  2. FDA Regenerative Medicine Advanced Therapy designation for P-BCMA CAR T-cell therapy. https://multiplemyelomahub.com/medical-information/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

Newsletter

Subscribe to get the best content related to multiple myeloma delivered to your inbox