Imugene Ltd (ASX:IMU, OTC:IUGNF) has been granted Orphan Drug Designation (ODD) by the United States Food and Drug Administration (FDA) for its novel oncolytic virotherapy, CF33-hNIS (VAXINIA), aimed at treating cholangiocarcinoma, a rare and aggressive bile duct cancer.
The FDA's Orphan Drug status is awarded to encourage the development of treatments for rare diseases that impact fewer than 200,000 people in the US.
The designation presents several benefits for Imugene, including tax credits, possible grant funding, waiver of specific fees and seven years of market exclusivity once VAXINIA receives FDA approval.
"Receiving Orphan Drug Designation from the FDA is a major milestone for us. It reflects the potential of VAXINIA to address the urgent need for new treatments for cholangiocarcinoma, a disease with limited therapeutic options," Imugene managing director and CEO Leslie Chong said.
"We are excited to continue advancing this program, which has already shown meaningful clinical responses in patients."
ODD is a big deal
Generally, speaking orphan drug designation is a big deal.
It offers a range of valuable incentives to support the development of treatments for rare diseases. As reflected by Imugene’s designation, benefits include tax credits for qualified clinical testing in humans, a waiver of the Prescription Drug User Fee—which currently approaches $3 million for new drug applications—and the potential for seven years of market exclusivity once the drug is approved.
In addition to these incentives, the Orphan Drug Act established the Orphan Product Grants Program, which provides financial assistance to promote the development of treatments targeting rare diseases or conditions. These measures are designed to encourage innovation and investment in areas of unmet medical need.
Promising results
Imugene's ODD application was backed by preclinical and clinical data from its ongoing Phase 1 MAST (Metastatic Advanced Solid Tumours) trial, which is evaluating the safety and effectiveness of VAXINIA. Early results from the trial are promising, with one cholangiocarcinoma patient experiencing a complete response and another achieving stable disease post-treatment.
The MAST trial is currently in its dose-escalation phase, where VAXINIA is administered either as a monotherapy or alongside checkpoint inhibitors. This trial has been expanded to include an additional cohort of 10 patients with bile duct cancers, including cholangiocarcinoma.
Cholangiocarcinoma, a rare cancer originating in the bile ducts, typically has a poor prognosis and demonstrates resistance to standard treatments such as chemotherapy and immunotherapy. Imugene's novel oncolytic virotherapy, VAXINIA, is engineered to selectively attack and eliminate cancer cells, while simultaneously triggering an immune response against the tumour.