This article provides a brief overview of FDA drug approvals in June 2025, highlighting new pharmaceutical stars bringing fresh hope to health and life:
Andembry (garadacimab-gxii) is an activated Factor XII (FXIIa) inhibitor (monoclonal antibody) approved for the prevention of hereditary angioedema attacks in patients aged 12 years and older. It is the first prophylactic treatment targeting FXIIa and the only therapy initially offering a once-monthly dosing regimen for all patients. Its citrate-free formulation allows for subcutaneous self-injection via autoinjector in 15 seconds or less.
Ibtrozi (taletrectinib) is a kinase inhibitor indicated for the treatment of adults with locally advanced or metastatic ROS1-positive NSCLC. Taletrectinib inhibits the tyrosine kinase ROS1, including resistance mutations, and also acts against TRK proteins (TRKA, TRKB, TRKC). ROS1 fusion proteins drive tumorigenesis by activating downstream signaling. Taletrectinib suppresses growth of cancer cells expressing ROS1 fusions and mutations. In mouse models with intracranial NSCLC xenografts expressing ROS1 fusions (including G2032R mutation), taletrectinib demonstrated antitumor activity.
Enflonsia (clesrovimab-cfor) is a fusion inhibitor targeting the RSV F protein. It is indicated for passive immunization to prevent lower respiratory tract disease due to RSV in newborns and infants entering their first RSV season. This long-acting monoclonal antibody provides rapid and sustained protection for five months, covering the entire RSV season (typically autumn to spring). A fixed 105 mg dose is used regardless of infant weight, simplifying clinical use.
Harliku (nitisinone) is a hydroxyphenylpyruvate dioxygenase inhibitor used to reduce homogentisic acid (HGA) in adults with alkaptonuria, a rare genetic metabolic disorder. It is the first approved treatment for AKU. Approval was based on a Phase 2 interventional study at NIH’s National Human Genome Research Institute showing long-term improvement in pain, energy, and physical function.
Yeztugo (lenacapavir) is an HIV-1 capsid inhibitor approved for PrEP to reduce the risk of sexually acquired HIV-1. It is the first HIV prevention method requiring only twice-yearly injections. FDA approval was based on two Phase 3 trials—PURPOSE 1 and PURPOSE 2—comparing lenacapavir to daily oral PrEP (Truvada or Descovy). Results showed a lower HIV infection rate with lenacapavir (0.10 vs. 0.93 per 100 person-years) and good safety. Recognized as a “Breakthrough Therapy” by the FDA in October 2024 and named Science’s #1 Breakthrough of 2024.
Zusduri (mitomycin) is a sustained-release hydrogel-based intravesical therapy for low-grade, intermediate-risk non–muscle-invasive bladder cancer (LG-IR-NMIBC). Formerly UGN-102, this formulation uses RTGel technology to prolong bladder exposure to mitomycin. It is instilled as a liquid and gels at body temperature. Administered via catheter in outpatient settings, Zusduri offers a non-surgical option.
Approval was based on the ENVISION trial (NCT05243550), a Phase 3, single-arm, open-label, multicenter study involving 240 patients with recurrent LG-NMIBC post-TURBT. Weekly 75 mg mitomycin was administered intravesically for six weeks. Among 223 evaluable patients, 78% achieved complete response at 3 months, with 79% maintaining response for at least 12 months. Most adverse events were mild to moderate; serious events occurred in 12% of patients, including urinary retention and urethral stricture.
Novel Drug Approvals for 2025. url: https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025
