Shilpa Medicare Limited's material subsidiary, Shilpa Biologicals, along with partner mAbTree Biologics, has received Orphan Drug Designation from the US Food and Drug Administration (USFDA) for its flagship investigational biologic aimed at treating rare blood cancers, the company announced on Wednesday, January 28. According to reports from CNBC TV18, the designation has been granted for a monoclonal antibody being developed for Essential Thrombocythemia and Polycythemia Vera - chronic myeloproliferative disorders with limited treatment options.
Orphan Drug status provides regulatory and commercial incentives to the company, including development support, tax credits and potential market exclusivity upon approval. As reported by CNBC TV18, the programme targets a previously underexplored immune-evasion pathway implicated in myeloproliferative neoplasm biology, with the biologic being described as a first-in-class, immunology-driven therapy designed to modify disease progression rather than only manage symptoms.
Sridevi, chief executive officer of Shilpa Biologicals Pvt Ltd, said this milestone marks a defining moment for Shilpa's biologics journey and validates the strength of our collaboration. According to CNBC TV18, she added that the company is moving rapidly towards clinical development with potential applications beyond rare blood cancers. Raj Andhuvan, chief executive officer of mAbTree Biologics AG, said the FDA designation validates the differentiated mechanism of the programme and could help establish a new therapeutic approach in rare blood cancers by targeting immune dysregulation.
Following the regulatory milestone, the companies plan to advance the biologic through IND-enabling studies, with the aim of initiating first-in-human clinical trials in patients with Essential Thrombocythemia and Polycythemia Vera. As reported by CNBC TV18, this represents a significant step towards bringing this novel treatment approach to patients with these rare and often difficult-to-treat blood disorders.
