LAVA Therapeutics (LVTX) Granted FDA Orphan Drug Designation for LAVA-051 for the Treatment of Chronic Lymphocytic Leukemia

LAVA Therapeutics N.V. (Nasdaq: LVTX), a clinical-stage biotechnology company, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for the company’s CD1d targeted GammabodyTM, LAVA-051, for the treatment of chronic lymphocytic leukemia (CLL). CLL is a form of leukemia characterized by progressive accumulation of abnormal lymphocytes in the peripheral blood, bone marrow and lymphoid tissues.

"We are excited to receive our first orphan drug designation from the FDA for LAVA-051, our most advanced product candidate from our off-the-shelf GammabodyTM platform that is designed to unlock the full anti-cancer potential of this specialized effector cell population,” said Stephen Hurly, president and chief executive officer, LAVA Therapeutics. “This designation will be helpful in enhancing our communication with the FDA on our development of LAVA-051. We are grateful to the FDA for highlighting the need for new and improved therapies to address the unmet needs in CLL.”

The GammabodyTM LAVA-051 is a bispecific antibody that recruits the immune system to attack CD1d-expressing cancer cells via the preferential activation of both Gamma Delta T cells and type 1 natural killer T (NKT) cells. Enrollment is underway in the company’s open-label, multi-center, Phase 1/2a clinical trial for the treatment of relapsed and/or refractory CLL, multiple myeloma (MM) and, later in the trial, acute myeloid leukemia (AML) (NCT04887259). Initiated in July 2021, the trial is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and preliminary antitumor activity of LAVA-051. Data from the Phase 1 dose escalation phase of the study are expected in the first half of 2022 with top line clinical data from the Phase 2a expansion cohorts expected in the second half of 2022.

About Orphan Drug Designation

The term orphan drug refers to pharmaceutical products developed for the prevention, diagnosis and treatment of rare diseases or conditions. In the U.S., an orphan disease is defined as a disease or condition with a U.S. prevalence of less than 200,000 people. Supporting the development and evaluation of new treatments for rare diseases is a key priority for the FDA. Since the Orphan Drug Act was passed in 1983, the U.S. government has provided incentives and policy support to encourage development of orphan drugs to meet medical unmet needs. This orphan drug designation from the FDA qualifies LAVA for various incentives related to the development of LAVA-051, including tax credits for qualified clinical trials, exemption from user fees and the potential for seven years of U.S. market exclusivity for the treatment of CLL.

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