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 Know AML.
Introducing
Now you can personalise
your AML Hub experience!
Bookmark content to read later
Select your specific areas of interest
View content recommended for you
Find out moreThe AML 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 AML Hub cannot guarantee the accuracy of translated content. The AML 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.
Bookmark this article
The FDA has granted an investigational chimeric antigen receptor (CAR) T-cell therapy, called MB-102, orphan drug designation. This means that the drug now has ‘orphan status’; those intended for effective and safe treatment of rare diseases affecting fewer than 200,000 people in the US, or affecting more than this, but is not expected to recover costs of developing and manufacturing the drug.
This CAR T therapy targets the CD123 antigen and was previously granted orphan drug designation in December 2018 for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). CD123 is widely expressed in the bone marrow cells of patients with myelodysplastic syndromes and hematological malignancies.
It is expected that a phase I/II multicenter trial will begin in the next few months, with a phase I first-in-human trial (NCT02159495) of MB-102 identifying that low doses of the experimental drug have resulted in complete responses in patients with AML. This dose-escalation trial currently being conducted will determine the safety and efficacy of the drug in patients with relapsed or refractory AML or BPDCN.
So far, no patients have experienced higher than grade III cytokine release syndrome or neurotoxicity, with both being reversible and well-managed.
The orphan drug designation allows the manufacturers to qualify for various incentives, such as credits for clinical trials, and if approved, seven years of market exclusivity.
Your opinion matters
Subscribe to get the best content related to AML delivered to your inbox