FDA Grants Orphan Drug Designation to BCMA-targeted Multiple Myeloma Therapy
The FDA has granted an orphan drug designation to the BCMA-targeted trispecific T-cell activating recombinant protein construct as treatment for patients with multiple myeloma.
The FDA has granted an orphan drug designation to the BCMA-targeted trispecific T-cell activating recombinant protein construct (TriTAC) as treatment for patients with multiple myeloma, according to an announcement from Harpoon Therapeutics, Inc.1 The agent is currently under exploration in a multicenter, open-label, phase 1/2 trial (NCT04184050), which was designed to examine the safety, tolerability, pharmacokinetics, and activity of HPN217 in patients with relapsed/refractory multiple myeloma.2
“Orphan drug designation for multiple myeloma represents a significant milestone in the development of HPN217 and recognizes its potential to address a significant unmet medical need for the patients suffering from this condition,” Jerry McMahon, PhD, president and chief executive of Harpoon Therapeutics, stated in a press release. “I am pleased with the clinical progress we are making with this program and we are planning to present interim data from the ongoing phase 1/2 dose-escalation trial later this year.”
BCMA is expressed on B lineage cells such as memory B cells, plasmablasts, and plasma cells.3 It is known that the BCMA ligands TNFSF13/APRIL and TNFSF13B/BAFF activate the MAP kinase and BcL-2/XL pathways to encourage proliferation and survival and that BCMA expression is upheld after plasma cell transformation into myeloma. As such, BCMA is under exploration as an important target for several therapies that are being developed to combat this disease.