FDA Updates

In December 2023, the FDA granted expanded indications for bempedoic acid and bempedoic acid plus ezetimibe in the treatment of primary hyperlipidemia.

Daratumumab and hyaluronidase-fihj in combination with other therapies was previously approved by the FDA in May 2020 for 8 indications in multiple myeloma.

Afami-cel had engineered t-cell receptor T-cells target MAGE-A4+ tumors, which are highly expressed in synovial sarcoma with HLA-A*02.

The designation offers support in developing potential new medicines, treatment, and diagnosis to prevent rare conditions, like EoE.

Less manufacturing time could be the difference between a patient receiving this life-saving therapy vs not.

Trastuzumab deruxtecan and elacestrant represent important new treatment options.

The approval marks dupilumab as the first and only FDA-approved medicine to treat pediatric patients with eosinophilic esophagitis.

According to the FDA review, patients with CKD receiving denosumab for osteoporosis who are on dialysis or have CKD-MBD are at the highest risk of severe hypocalcemia.

The treatment is for patients with KRAS G12C-mutant metastatic NSCLC who have previously received at least 1 systemic therapy and were not treated with a KRAS G12C inhibitor.

Previously, IgG-hyaluronidase recombinant was approved by the FDA in 2014 for the treatment of primary immunodeficiency in adult patients and has expanded to include some pediatric patients.

The approval was based on the results of a phase 3 trial that found an improvement in progression-free survival among individuals treated with pembrolizumab.

BrainSee creates a new standard for progression prediction in Alzheimer disease, marking a step forward in brain health management with non-invasive screening.

The accepted sBLA comes after phase 3 clinical trial results which demonstrated better overall survival, progression-free survival, and confirmed overall response rate than chemotherapy in patients with cervical cancer.

Teneo Excimer Laser Platform is a new laser assistance in situ keratomileusis for vision correction surgery for those who have myopia and myopic astigmatism.

Berdazimer topical gel, 10.3% (Zelsuvmi; Ligand Pharmaceuticals Inc) has been approved for the treatment of molluscum contagiosum in individuals aged 1 year and older.

Pharmacists' expertise is crucial in educating patients about regulatory changes, helping them navigate alternative formulations, and providing insights into evidence-based choices.

Adalimumab-aqvh (Yusimry; Coherus Biosciences) entered the market in July 2023, following approval by the FDA in December 2021.

These drugs have interesting mechanisms, unique use, or may support expanded indications in the future.

This approval makes birch triterpenes the first treatment for wounds associated with junctional epidermolysis bullosa to be approved by the FDA.

If approved, V116 would offer significant improvements in safety and effectiveness for the prevention of pneumococcal disease.

Previously, garadacimab was granted an orphan drug designation as a therapy for hereditary angioedema by both the FDA and EMA.

The approval marks the first for an alternative to platinum-containing chemotherapy.

Belzutifan (Welireg; Merck) demonstrated superior progression-free survival compared to everolimus in advanced renal cell carcinoma.

The study will assess the use of the drug to treat impaired social motivation, or asociality, which is a difficult-to-treat symptom of schizophrenia that can cause significant functional impairment.

Officials from the FDA have granted Geron Corporation’s imetelstat fast track designation for adult patients with intermediate-2 or high-risk myelofibrosis (MF) whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment.