Shilpa Biologicals and mAbTree Biologics secure ODD from USFDA

Marks significant milestone for breakthrough biologic to treat rare blood cancers

Shilpa Biologicals (part of the Shilpa Medicare Group) and partner mAbTree Biologics AG have achieved a major regulatory milestone, receiving Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for their flagship investigational monoclonal antibody, targeting rare, chronic blood cancers.

This first-in-class biologic is being developed to treat Essential Thrombocythemia (ET) and Polycythemia Vera (PV), offering a potential new therapy that modifies disease progression rather than just managing symptoms.

This novel checkpoint inhibitor may also have potential in additional cancer indications, including lung cancer and head and neck squamous cell carcinoma.

Securing Orphan Drug Designation recognizes both the seriousness of ET and PV and the potential of this first-in-class, immunology-driven biologic to modify disease progression rather than only managing symptoms. It also provides regulatory and commercial 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 Dr. Sridevi, Chief Executive Officer of Shilpa Biologics Pvt. Ltd. “The FDA’s recognition of our flagship biologic underscores the quality of the science and our ability to translate innovation into globally relevant clinical programs. We are advancing rapidly toward clinical development and see strong potential beyond rare blood cancers.”

Patients with ET and PV often require lifelong treatment. Existing therapies—including aspirin, interferon-alpha, hydroxyurea, and JAK inhibitors—have improved disease management; however, a significant proportion of patients become refractory or intolerant over time, reinforcing the need for therapies that directly address disease biology.

“Receiving Orphan Drug Designation from the FDA is a strong validation of the differentiated mechanism behind this program,” said Raj Andhuvan, Chief Executive Officer 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 biologic targets a previously underexplored immune-evasion pathway implicated in Myeloproliferative neoplasms biology, offering the potential for durable disease control and a clearly differentiated clinical profile.

Following this regulatory milestone, Shilpa Biologicals and mAbTree Biologics AG will advance the program through IND-enabling studies, with the objective of initiating first-in-human clinical trials in patients with ET and PV.