USFDA Grants Orphan Drug Status to Shilpa-mAbTree Biologic for Rare Blood Cancers
New Delhi: Shilpa Biologicals Pvt Ltd, part of the Shilpa Medicare Group, along with Switzerland-based mAbTree Biologics AG, has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (USFDA) for an investigational monoclonal antibody being developed for the treatment of Essential Thrombocythemia (ET) and Polycythemia Vera (PV).
ET and PV are rare, chronic myeloproliferative neoplasms characterised by abnormal blood cell production and limited long-term treatment options. The investigational biologic is designed to target disease-driving immune mechanisms and is intended to address disease progression rather than only symptom control.
The Orphan Drug Designation is granted to therapies intended to treat rare diseases affecting fewer than 200,000 patients in the United States. The designation provides regulatory incentives, including protocol assistance, tax credits for clinical development, and seven years of market exclusivity in the US following approval.
Patients with ET and PV typically require lifelong treatment. While currently available therapies—such as aspirin, interferon-alpha, hydroxyurea, and JAK inhibitors—have improved disease management, a significant proportion of patients develop resistance or intolerance over time, highlighting the need for novel therapeutic approaches.
According to the companies, the monoclonal antibody targets an immune-evasion pathway implicated in the underlying biology of myeloproliferative neoplasms. Following the ODD grant, Shilpa Biologicals and mAbTree Biologics plan to advance the program through IND-enabling studies, with the aim of initiating first-in-human clinical trials in patients with ET and PV.
