The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to an investigational monoclonal antibody being co-developed by Shilpa Biologicals and mAbTree Biologics.
According to an announcement made by the pair, the flagship breakthrough biologic is being developed for the treatment of Essential Thrombocythemia (ET) and Polycythemia Vera (PV)—rare, chronic blood cancers with significant unmet medical need.
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 with time, highlighting the need for therapies that directly address disease biology.
The investigational biologic targets a previously underexplored immune-evasion pathway implicated in the biology of Myeloproliferative neoplasms, offering the potential for durable disease control and a clearly differentiated clinical profile.
It may also have potential in additional cancer indications, including lung cancer and head and neck squamous cell carcinoma.
Obtaining the ODD "underscores the seriousness of ET and PV," the companies said in a statement, as well as the potential of this first-in-class, immunology-driven biologic to "modify disease progression rather than merely manage its symptoms."
Additionally, it offers the duo regulatory and commercial benefits, such as 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, CEO of Shilpa Biologics.
The FDA's recognition of our flagship biologic underscores the quality of the science and our ability to translate innovation into globally relevant clinical programmes.
"We are advancing rapidly toward clinical development and see strong potential beyond rare blood cancers."
"Receiving Orphan Drug Designation from the FDA is a strong validation of the differentiated mechanism behind this programme," added Raj Andhuvan, CEO of mAbTree Biologics.
By targeting immune dysregulation—now recognised 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.
Following this regulatory milestone, Shilpa Biologicals and mAbTree Biologics say that they will advance the programme through IND-enabling studies, with the objective of initiating first-in-human clinical trials in patients with ET and PV.