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 MDS Alliance.

The MDS 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
  TRANSLATE

The MDS 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 MDS Hub cannot guarantee the accuracy of translated content. The MDS 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

Briquilimab granted orphan drug designation by the European Commission

Jan 5, 2023
Share:
Learning objective: After reading this article, learners will be able to cite a new development in the treatment of MDS .

On January 4, 2023, briquilimab, a monoclonal antibody targeting the CD117 receptor, was granted orphan drug designation by the European Commission, as a conditioning treatment prior to hematopoietic stem cell transplantation (HSCT).1 Briquilimab received orphan drug designation from the U.S Food and Drug Administration (FDA) in June 2022.1

As previously reported by the AML Hub, briquilimab is currently being investigated as a conditioning treatment prior to HSCT in patients with acute myeloid leukemia and myelodysplastic syndromes (MDS).1 Briquilimab was found to have a tolerable safety profile, with full donor chimerism in all 24 patients with acute myeloid leukemia or MDS. There were no reports of treatment-related significant adverse events, classical acute Grade II–IV graft-versus-host disease, or transplant mortality within 100 days. Briquilimab is also being assessed as a primary therapy for patients with low-risk MDS and as a conditioning therapy prior to HSCT for patients with severe combined immunodeficiency.1

  1. Jasper Therapeutics. Jasper Therapeutics announces European Union orphan drug designation for briquilimab as a conditioning treatment for patients prior to receiving a stem cell transplant. https://ir.jaspertherapeutics.com/news-releases/news-release-details/jasper-therapeutics-announces-european-union-orphan-drug. Published Jan 4, 2023. Accessed Jan 5, 2023.

Your opinion matters

Which is a global phase III trial for MDS that utilizes a biomarker for patient selection? ENHANCE (magrolimab), SELECT-MDS-1 (tamibarotene), VERONA (venetoclax), or STIMILUS-MDS3 (sabatolimab)?
0 votes - 10 days left ...

Newsletter

Subscribe to get the best content related to MDS delivered to your inbox