The U.S. Food and Drug Administration (FDA) has recently approved several new therapies and expanded the use of an existing diagnostic test, addressing unmet needs in oncology, rare genetic disorders, and infectious diseases.
Bizengri Approved for NRG1 Fusion-Positive Cancers
On December 6, 2024, the FDA granted accelerated approval to Bizengri (zenocutuzumab-zbco, Merus N.V.) for adult patients with advanced, unresectable, or metastatic non-small cell lung cancer (NSCLC) or pancreatic adenocarcinoma harboring a neuregulin 1 (NRG1) gene fusion who have experienced disease progression on or after prior systemic therapy. This approval marks the first FDA approval of a systemic therapy specifically targeting NRG1 gene fusions in these cancers.
The approval was based on clinical trial data demonstrating efficacy in patients with NRG1 fusion-positive cancers. Common adverse reactions reported in the pooled safety population (≥10%) included diarrhea, musculoskeletal pain, fatigue, nausea, infusion-related reactions, dyspnea, rash, constipation, vomiting, abdominal pain, and edema.
Ryoncil Approved for Steroid-Refractory Acute Graft Versus Host Disease
On December 20, 2024, the FDA approved Ryoncil (remestemcel-L-rknd), an allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy, for the treatment of steroid-refractory acute graft versus host disease (SR-aGVHD) in pediatric patients aged 2 months and older. This is the first FDA-approved MSC therapy.
The most common nonlaboratory adverse reactions (incidence ≥20%) were viral infectious disorders, bacterial infectious disorders, infection – pathogen unspecified, pyrexia, hemorrhage, edema, abdominal pain, and hypertension.
OraQuick HIV Self-Test Approved for Adolescent Use
The FDA approved a premarket approval application (PMA) supplement for the OraQuick Human Immunodeficiency Virus (HIV) Self-Test, expanding its approved age range to include individuals aged 14 to 17 years. This approval marks the first over-the-counter HIV test available for adolescents, aiming to improve early detection of HIV in this population.
The OraQuick HIV Self-Test is a single home-use test designed to detect antibodies to Human Immunodeficiency Virus Type 1 (HIV-1) and Type 2 (HIV-2) in oral fluid specimens. The test is not intended for use with other types of specimens, and individuals are advised to seek confirmatory testing in a medical setting.
Tryngolza Approved for Familial Chylomicronemia Syndrome
Tryngolza (olezarsen) received FDA approval on December 20, 2024, for use in conjunction with diet to reduce triglycerides (TG) in adults with familial chylomicronemia syndrome (FCS). FCS is a rare genetic disorder that impairs the body's ability to break down fats in the bloodstream, leading to severely elevated TG levels. This approval is significant as Tryngolza represents a first-in-class therapy with a novel mechanism of action for treating FCS.
The most common adverse reactions associated with Tryngolza were injection site reactions, decreased platelet count, and arthralgia.
