On September 18, 2024 Imugene Limited (ASX:IMU), a clinical-stage immuno‐ oncology company, reported that it has received Orphan Drug Designation (ODD) from the United States Food and Drug Administration (FDA) for its novel oncolytic virotherapy CF33-hNIS (VAXINIA), for the treatment of cholangiocarcinoma, a rare and aggressive form of bile tract cancer (Press release, Imugene, SEP 18, 2024, https://mcusercontent.com/e38c43331936a9627acb6427c/files/d4c82d92-18f3-59f9-598e-a451d46a7ffe/Imugene_Receives_Orphan_Drug_Designation_for_VAXINIA.pdf [SID1234646708]).
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The FDA grants Orphan Drug status to promote development of drugs for rare diseases that affect fewer than 200,000 people in the United States. This designation provides Imugene with a range of incentives, including tax credits, potential grant funding, waiver of certain administrative fees, and seven years of market exclusivity upon FDA approval.
Imugene’s application was supported by preclinical and clinical data from its ongoing Phase 1 MAST (Metastatic Advanced Solid Tumours) trial, which is investigating the safety and efficacy of VAXINIA. Promising results have already been observed, including one patient with cholangiocarcinoma achieving a complete response and another achieving stable disease after treatment with VAXINIA.
The MAST trial, which involves delivering the oncolytic virus either as a monotherapy or in combination with checkpoint inhibitors, is currently in the dose-escalation phase. This has recently been expanded into a further cohort of 10 patients with bile tract cancers, including cholangiocarcinoma.
Imugene’s Managing Director & CEO, Leslie Chong, commented: "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. We are excited to continue advancing this program, which has already shown meaningful clinical responses in patients."
Cholangiocarcinoma is a rare malignancy arising from the bile ducts, which has a poor prognosis and is often resistant to conventional treatments, including chemotherapy and immunotherapy. Imugene’s innovative oncolytic virotherapy, VAXINIA, is designed to selectively target and destroy cancer cells, while also stimulating an immune response against the tumour.