By: IPP Bureau
Last updated : January 30, 2026 7:17 pm
The pioneering monoclonal antibody is for Essential Thrombocythemia (ET) and Polycythemia Vera (PV), rare chronic blood cancers with limited treatment options
Shilpa Biologicals, part of the Shilpa Medicare Group, and mAbTree Biologics AG have announced a major regulatory breakthrough: the US FDA has granted Orphan Drug Designation (ODD) for their rare blood cancer therapy.
The pioneering monoclonal antibody is for Essential Thrombocythemia (ET) and Polycythemia Vera (PV), rare chronic blood cancers with limited treatment options.
The designation highlights both the seriousness of ET and PV and the potential of this first-in-class, immunology-driven biologic to modify disease progression, not just manage symptoms. It also offers critical incentives, including development support, tax credits, and market exclusivity upon approval.
“This milestone marks a defining moment for Shilpa’s Biologics journey and validates the strength of our collaboration with mAbTree Biologics,” said Sridevi, CEO of Shilpa Biologicals. “The FDA’s recognition of our flagship biologic underscores the quality of the science and our ability to translate innovation into globally relevant critical care programs. We are advancing rapidly toward clinical development and see strong potential beyond rare blood cancers.”
Current treatments for ET and PV—ranging from aspirin and interferon-alpha to hydroxyurea and JAK inhibitors—help manage symptoms but often fail over time, leaving patients refractory or intolerant. The need for therapies that directly target disease biology remains urgent.
“Receiving Orphan Drug Designation from the FDA is a strong validation of the differentiated mechanism behind this program,” said Raj Andhuvan, CEO of mAbTree Biologics AG. “By targeting immune dysregulation—now recognized as a central driver of disease persistence in myeloproliferative neoplasms—this biologic has the potential to establish a new therapeutic paradigm in rare blood cancers.”
The investigational therapy attacks a previously underexplored immune-evasion pathway, offering durable disease control and a uniquely differentiated clinical profile. Following this milestone, Shilpa Biologicals and mAbTree Biologics will advance the program through IND-enabling studies, aiming to launch first-in-human clinical trials in patients with ET and PV.