LAVA Therapeutics Gets FDA Orphan Drug Designation for Leukemia Treatment

  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.

https://finance.yahoo.com/news/lava-therapeutics-receives-fda-orphan-110000871.html