Regulatory Milestone for Shilpa Biologicals and mAbTree Biologics
Shilpa Biologicals Pvt. Ltd., part of the Shilpa Medicare Limited Group, has achieved a significant regulatory milestone in collaboration with mAbTree Biologics AG. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to their investigational monoclonal antibody, a breakthrough biologic being developed to treat rare blood cancers such as Essential Thrombocythemia (ET) and Polycythemia Vera (PV).
These conditions are chronic, rare cancers with limited treatment options and significant unmet medical need. Therefore, the designation highlights the potential of this innovative therapy to address a critical gap in care.
Potential Applications Beyond Rare Blood Cancers
In addition to ET and PV, the novel biologic may also demonstrate therapeutic potential in other cancer indications. Researchers are exploring its possible use in conditions such as Lung Cancer and Head and Neck Squamous Cell Carcinoma.
As a checkpoint inhibitor, the therapy uses an immunology-driven approach to target disease mechanisms. Consequently, it aims not only to manage symptoms but also to potentially modify disease progression.
Benefits of Orphan Drug Designation
The Orphan Drug Designation acknowledges both the seriousness of ET and PV and the promise of this first-in-class biologic. Moreover, the designation offers several regulatory and commercial advantages, including development support, tax incentives, and potential market exclusivity following approval.
Commenting on the achievement, Dr. Sridevi, Chief Executive Officer of Shilpa Biologicals Pvt. Ltd., said the milestone represents an important moment in the company’s biologics journey. She noted that the FDA’s recognition validates the strength of the collaboration with mAbTree Biologics and highlights the scientific quality behind the program. She further added that the company is moving rapidly toward clinical development while exploring broader applications beyond rare blood cancers.
Need for Innovative Treatments in ET and PV
Patients living with Essential Thrombocythemia and Polycythemia Vera often require lifelong treatment. Current therapies—including Aspirin, Interferon‑alpha, Hydroxyurea, and JAK inhibitors—have improved disease management. However, a considerable number of patients eventually become resistant or intolerant to these treatments.
Therefore, the development of therapies that directly address the underlying disease biology remains a priority.
Targeting Immune Dysregulation in Blood Cancers
As per the press release, Raj Andhuvan, Chief Executive Officer of mAbTree Biologics AG, described the FDA designation as strong validation of the biologic’s differentiated mechanism of action. According to him, the therapy targets immune dysregulation, which researchers increasingly recognize as a key driver of disease persistence in Myeloproliferative Neoplasms.
By addressing this immune-evasion pathway, the investigational therapy may offer durable disease control and a distinct clinical profile.
Next Steps Toward Clinical Development
Following this regulatory milestone, Shilpa Biologicals and mAbTree Biologics will advance the program through Investigational New Drug (IND)-enabling studies. The companies aim to initiate first-in-human clinical trials in patients with ET and PV in the near future.
Industry Engagement at BIO-Europe Spring 2026
Meanwhile, Shilpa Biologicals will participate in BIO‑Europe Spring 2026, scheduled to take place in Lisbon from March 23–25. During the event, the company expects strong interest from emerging biotechnology firms in its contract development and manufacturing organization (CDMO) services as it continues expanding partnerships in biologics discovery and development.