FDA Updates

An oral dose of 0.6 mg per 5 mL of colchicine could aid individuals who have trouble with swallowing pills.

The breakthrough designation is supported by clinical data from ongoing phase 1/2 trials.

Multiple phase 3 clinical trials demonstrated dupilumab’s (Dupixent; Regeneron, Sanofi) effectiveness in reducing hives and itch in patients with chronic spontaneous urticaria compared with placebo.

Some patients with aplastic anemia treated with CK0801 achieved durable transfusion independence lasting over 3 years.

The safety profile of diazepam nasal spray among pediatric patients was consistent with older patients, supporting its use in this age group.

The designation was supported based on data from the randomized, double-blind, active-controlled phase 3 interchangeability study.

The new indication expands administration methods for the combination of efgartigimod alfa and hyaluronidase-qvfc, which are already approved for the treatment of some immune-related conditions.

Patients with unresectable or metastatic hepatocellular carcinoma (HCC) receiving this regimen had favorable median overall survival (23.7 months) compared with standard of care (20.6 months).

This approval marks bevacizumab-nwgd as the sixth reference product to bevacizumab (Avastin).

The approval is for those with metastatic disease or in whom surgical resection is likely to result in severe morbidity, have no satisfactory alternative treatments, or have progressed following treatment.

Data show that the system is also the most accurate continuous glucose monitor (CGM).

The approval was based on efficacy and safety data from a randomized, 3-arm, open-label phase 3 CHECKMATE-8HW trial.

The new designations span both warm autoimmune hemolytic anemia and IgG4-related disease, 2 rare, immune-mediated conditions that burden patients due to a lack of available treatment options.

BIIB080 is designed to target microtubule-associated protein tau mRNA, aimed to reduce the production of tau protein.

This action makes inebilizumab the first and only FDA-approved treatment for adults with immunoglobulin G4-related disease (IgG4-RD).

No manufacturing or safety issues were identified, but the FDA stated the new drug application did not demonstrate efficacy in adequate and well-controlled studies.

Positive results from an interim analysis of the phase 3 ALIGN study bolstered atrasentan towards receiving accelerated approval for patients with immunoglobulin A nephropathy, a rare condition that can cause kidney failure.

The Auto-Pure 2400 system combines liquid handling and magnetic cell isolation for efficient latent tuberculosis testing.

A phase 1/2 clinical trial will provide crucial data on the vaccine's tolerability and pave the way for future development and potential widespread use.

The antibody-drug conjugate is currently undergoing evaluation in a phase 1 trial that enrolled patients with pancreatic cancer and squamous non-small cell lung cancer (NSCLC).

Currently, BEAM-302 is undergoing a phase 1/2 clinical trial to evaluate its efficacy in patients with alpha-1 antitrypsin deficiency (AATD) who have lung disease and liver disease.

Approval of durvalumab is in combination with gemcitabine and cisplatin as neoadjuvant treatment for adults with muscle invasive bladder cancer.

Approval of fitusiran, a small interfering RNA drug, was found effective in patients with hemophilia regardless of inhibitor status.

The biosimilar is approved for all indications of the reference products, Prolia and Xgeva.

The approval fills a treatment gap for patients with this rare disease, which can severely diminish quality of life.

Patients achieved statistically significant benefits in progression-free survival and overall response rates.

Gepotidacin is indicated for female patients who are at least 12 years of age and weigh at least 40 kg with uncomplicated urinary tract infections (uUTIs) caused by certain microorganisms.

If approved, this novel monoclonal antibody therapy could become a standard of care emergency option for patients with acute myocardial infarction, which affects millions in the United States and across the world.

Sjögren's disease is one of the most common chronic autoimmune diseases that affects 4 million individuals around the world.