Shilpa Biologicals Wins FDA Orphan Drug Status for Cancer Therapy

Shilpa Biologicals, part of Shilpa Medicare, along with partner mAbTree Biologics AG, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration for its investigational monoclonal antibody designed to treat rare blood cancers. The FDA Orphan Drug Designation marks a key regulatory step for the company’s flagship biologic therapy.

The investigational treatment targets Essential Thrombocythemia (ET) and Polycythemia Vera (PV), two rare chronic blood cancers classified under Myeloproliferative Neoplasms. Unlike current therapies that mainly control symptoms, the biologic is designed to modify the underlying disease process and potentially slow progression.

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The therapy works as a novel checkpoint inhibitor aimed at a previously underexplored immune-evasion pathway involved in the persistence of these cancers. Researchers believe this mechanism could lead to more durable disease control and open new possibilities in oncology. Beyond ET and PV, the program may also expand into other cancers such as Lung Cancer and Head and Neck Squamous Cell Carcinoma.

“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 Orphan Drug Designation highlights the urgent need for new treatments. Patients with ET and PV often depend on long-term therapies such as aspirin, interferon-alpha, hydroxyurea, and JAK inhibitors, but many eventually become resistant or unable to tolerate them.

“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, said Raj Andhuvan, Chief Executive Officer of mAbTree Biologics AG.”

Following this milestone, the companies plan to complete IND-enabling studies and move toward first-in-human clinical trials for patients with ET and PV.