FDA Grants Orphan Drug Designation to BVX001 in AML


BVX001 has received orphan drug designation from the FDA for the treatment of patients with acute myeloid leukemia.



The FDA has granted orphan drug designation to BVX001 as a potential treatment option for patients with acute myeloid leukemia (AML). An Initial Targeted Engagement for Regulatory Advice (INTERACT) meeting with the FDA Center for Drug Evaluation and Research has also been completed to gain guidance regarding questions related to the pharmacology; toxicology; and chemistry, manufacturing, and controls of BVX001 to prepare for the filing of an investigational new drug application.1

“The orphan drug designation grant for our lead asset BVX001 represents important United States regulatory progress for BiVictriX [Therapeutics], highlighting the significant unmet need for novel therapies targeting AML and providing us with greater market exclusivity potential,” Tiffany Thorn, founder and chief executive officer of BiVictriX, stated in a news release. “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–investigational new drug application, and to present further efficacy and safety data at a key hematology conference in the second half of 2024.”

“Early engagement with the FDA for novel therapeutic approaches is key, and I am delighted with our progress to date,” Michael Kauffman, MD, PhD, chairman of BiVictriX, added in the news release. “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 orphan drug designation. I am confident that BVX001 has unique and novel attributes that will support its advancement to the clinic. This timely FDA guidance and orphan drug designation will expedite BVX001’s development, as we look to work closely with the FDA and other regulatory agencies.”

BVX001, a novel, first-in-class CD33/CD7-directed antibody-drug conjugate, generated prolonged overall survival (OS) rates in preclinical AML models in a study comparing BVX001 at 10 mg/kg twice weekly vs the highest accepted short-term dose of cytarabine for fit patients with AML (HiDAC) vs an untreated control arm that received the vehicle only.2 After a 28-day dosing period, the median OS was 129 days with BVX001 vs 91 days with HiDAC vs 57 days in the untreated control arm, translating to a 126% median OS advantage with BVX001 vs the untreated control arm and a 42% median OS advantage with BVX001 vs HiDAC. Notably, the preclinical model represented a model of AML that was more challenging to treat than AML that is encountered in the clinic, as it contained more cells to drive cancer progression than the number of leukemia-initiating cells or leukemia stem cells found in patients with AML.

Previously, findings from the study showed that development leads of BVX001, BVX001-1, and BVX001-2, when administered at doses of 10 mg/kg twice weekly, 10 mg/kg once weekly, and 3 mg/kg twice weekly, were associated with a statistically significant tumor growth inhibition of greater than 87% at day 28 compared with the untreated control arm in preclinical murine models of AML (P < .001).3 At the twice-weekly 10 mg/kg dose, BVX001-1 and BVX001-2 induced statistically significant tumor volume regressions of 93% and 89%, respectively (P < .001). Furthermore, all dose levels of BVX001 were well tolerated.


  1. BiVictriX announces FDA orphan drug designation and completion of FDA INTERACT meeting for BVX001 in the treatment of acute myeloid leukemia. News release. BiVictriX Therapeutics plc. April 17, 2024. Accessed April 17, 2024. https://bivictrix.com/news/bivictrix-announces-fda-orphan-drug-designation-and-completion-of-fda-interact-meeting-for-bvx001-in-the-treatment-of-acute-myeloid-leukaemia/
  2. Extended survival rates reported with BVX001 in a pre-clinical model of acute myeloid leukemia. News release. BiVictriX Therapeutics plc. October 12, 2023. Accessed April 17, 2024. https://bivictrix.com/news/extended-survival-rates-reported-with-bvx001-in-a-pre-clinical-model-of-acute-myeloid-leukaemia/
  3. BiVictriX nominates clinical candidate for BVX001. News release. BiVictriX Therapeutics plc. June 6, 2023. Accessed April 17, 2024. https://bivictrix.com/news/bivictrix-nominates-clinical-candidate-for-bvx001/
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