FDA Updates

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 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.

Afami-cel was approved in conjunction with MAGE-A4 IHC 1F9 pharmDx, a diagnostic tool that can identify patients eligible to receive the treatment for synovial sarcoma.

The diagnostic tool can aid the identification of patients with synovial sarcoma who may be eligible for treatment with newly approved afamitresgene autoleucel.

This is the first and only on-demand, virus-inactivated, human plasma-derived concentration option that is indicated for this approval.

The RUBY trial will continue and analyze the overall population survival after treatment with the drug combination.

The drug previously received breakthrough therapy designation for newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia that is in chronic phase.

The designation is based on animal model studies that have demonstrated superior efficacy in comparison to sildenafil.

If approved, the drug would be the first in a new class of medications to treat acute pain in over 20 years.

The decision is based on positive results from the PERSEUS trial.

The approval streamlines treatment administration to eliminate compounding steps, reduce preparation time, and enhance safety for clinicians.

The approval marks the first oral immunotherapy treatment to mitigate allergic reactions, including anaphylaxis.