FDA Updates

Propofol has a history of chronic shortage due to supply chain constraints.

Vimseltinib (Deciphera Pharmaceuticals) has a Prescription Drug User Fee Act goal date of February 17, 2025.

The combination is the first and only multi-targeted chemotherapy-free regimen that demonstrated superiority compared with osimertinib for non–small cell lung cancer (NSCLC).

The highly potent, selective URAT1 inhibitor may help reduce serum uric acid levels and treat clinically visible tophi.

Fam-trastuzumab deruxtecan-nxki has received 4 breakthrough therapy designations, including the latest approval.

Currently, the drug will be studied in the SKYBRIDGE (NCT05652686) trial, a first-in-human, phase 1/2, open-label, dose-escalation and expansion study.

The OTC antibody test can detect current or past infection but should be followed by additional testing to confirm diagnosis.

The hemostatic gel is plant-based and designed to stop and control bleeding within seconds after applied to wound at the point of care.

Durvalumab is a human monoclonal antibody that targets and binds to PD-L1 to interrupt tumor immune-evasion tactics.

The decision is based positive data from the Deltacel-01 clinical trial.

AC699 is being evaluated for patients with estrogen receptor-positive (ER+), human epidermal growth factor receptor 2 negative (HER2–), estrogen receptor 1-mutated advanced or metastatic breast cancer with disease progression on or after at least 1 line of endocrine-based therapy.

The decision is based on clinical findings demonstrating seladelpar’s ability to effectively regulate of alkaline phosphatase values in patients.

The treatment is indicated to improve and maintain visual acuity in individuals with neovascular age-related macular degeneration.

Nemolizumab is the first approved monoclonal antibody specifically inhibiting the signaling of IL-31, which drives disease mechanisms in prurigo nodularis.

The autologous, fully human CD19 chimeric antigen receptor T-cell product, KYV-101, is being studied for the treatment of patients with progressive myasthenia gravis.

The commencement of the phase 1 clinical trial of SAT-3247 is expected in Q3 of 2024.

Previously, an independent advisory panel for the FDA rejected the use of MDMA-assisted therapy for posttraumatic stress disorder (PTSD), citing concerns of the reliability of the clinical trial data.

neffy is an epinephrine nasal spray for the emergency treatment of allergic reactions in adults and pediatric patients.

The FDA also granted rare pediatric disease designation for the drug.

The injection adds an additional option to address emergency known or suspected opioid overdoses.

The indication is for the reduction of proteinuria in adult patients who have primary immunoglobulin A nephropathy and are at risk of disease progression.

Denileukin diftitox-cxdl is the only therapy approved for cutaneous T-cell lymphoma that targets IL-2 receptors in malignant T-cells and Tregs.

Carbidopa and levodopa extended-release capsules are a novel, oral formulation that combine immediate-release granules and extended-release pellets.

This marks the first FDA-approved systemic therapy for patients with grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 (IHD1) or IDH2 mutation.

Sunobinop binds to and activate nociception/orphanin-FQ peptide receptor, which is a known therapeutic target for substance use disorder.

The drug also receives orphan drug designation for the treatment of pancreatic neuroendocrine tumors and a Prescription Drug User Fee Act target action date of April 3, 2025.

In the first half of 2024, the FDA approved 23 novel drugs for conditions including alopecia areata, Alzheimer disease, small cell lung cancer, bladder cancer, and more.

Afamitresgene autoleucel (afami-cel) is a novel cell therapy for the treatment of patients with synovial sarcoma.