BiVictriX Therapeutics PLC (AIM:BVX, OTC:BVTXF) told investors that the US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to BVX001, the company’s potential treatment for Acute Myeloid Leukemia (AML).
The designation recognises BVX001’s potential to address a significant unmet medical need, the company highlighted.
Additionally, the company said it has successfully concluded an Initial Targeted Engagement for Regulatory Advice (INTERACT) meeting with the FDA, which gave favourable guidance aligning with BiVictriX’s developmental strategy for BVX001.
"The ODD grant for our lead asset BVX001 represents important US regulatory progress for BiVictriX, highlighting the significant unmet need for novel therapies targeting AML and providing us with greater market exclusivity potential,” chief executive Tiffany Thorn said in a statement.
“The INTERACT forum gave us clear and timely guidance from the FDA on BVX001, and we consider the feedback to be very positive. With strong alignment between our future development plans and the comments from the FDA reviewers, it is an exciting time for the company.
“We continue to advance our preclinical data package for BVX001, with plans to submit a pre-IND application, and to present further efficacy and safety data at a key Haematology Conference in the second half of 2024."
BiVictriX chair Michael Kauffman added: “Early engagement with the FDA for novel therapeutic approaches is key, and I am delighted with our progress to date.
“Our ability to qualify for an INTERACT meeting underscores the novel and compelling approach we are taking with our scientific platform to develop truly differentiated therapies in areas of high unmet need, as demonstrated by our ODD.
“I am confident that BVX001 has unique and novel attributes that will support its advancement to the clinic. This timely FDA guidance and ODD will expedite BVX001's development, as we look to work closely with the FDA and other regulatory agencies."
Orphan Drug Designation by the FDA offers benefits such as tax credits and market exclusivity post-approval, which could significantly aid BiVictriX in navigating the path towards commercializing BVX001.