FDA Updates

This update follows May 2021 guidelines which recommended patients with primary biliary cholangitis (PBC) with advanced cirrhosis discontinue obeticholic acid.

The combination vaccine candidates will be evaluated in 2 separate phase 1/2 clinical trials.

PLT012 demonstrates its potential against multiple tumors with unmet medical needs.

Two studies found a 2-bag regimen of N-acetylcysteine (NAC) resulted in fewer and shorter delays in treatment as well as decreases in cutaneous non-allergic anaphylactoid reactions.

If approved, the treatment will be the first and only once-daily, oral selective somatostatin receptor type 2 nonpeptide agonist available for acromegaly.

As experts in medication management and health education, pharmacists can work with the administration to promote effective strategies that reduce misinformation and improve public health outcomes.

Durvalumab could offer further treatment for individuals with muscle-invasive bladder cancer.

Fabry disease is a rare lysosomal disorder that causes excessive deposition of lipids in tissues.

Zenocutuzumab-zbco is a bispecific antibody that targets and binds to HER2 and HER3.

Sacituzumab tirumotecan (sac-TMT; Kelun Pharmaceutical, Merck) is currently being studied to treat non-small cell lung cancer (NSCLC) in the TroFuse-004 and TroFuse-009 trials.

E-cigarette manufacturers claim the products aid in smoking cessation for adults without the risk of adolescent usage.

If approved, the new indications include children 6 years and older with moderate to severe plaque psoriasis and children 5 years and older with active juvenile psoriatic arthritis.

The biosimilar is approved for the treatment of Crohn disease, ulcerative colitis, plaque psoriasis, and psoriatic arthritis.

The approval is based on clinical studies that demonstrate the management of heart rate with minimal reductions to blood pressure.

Oral phenylephrine is a common ingredient in OTC products, but its clinical efficacy is questionable. The FDA is considering removing it from the market.

This is the first oral liquid form of imatinib (Imkeldi; Shorla Oncology) to be approved to treat cancers.

Acoramidis is a novel, highly potent transthyretin stabilizer.

Familial chylomicronemia syndrome (FCS) currently has no FDA-approved treatment and is associated with an increased risk of acute pancreatitis.

If approved, RAG-21 can offer improved outcomes in patients with amyotrophic lateral sclerosis (ALS), particularly those with aggressive subtypes.

As a rare and serious pediatric disorder, Stargardt disease can cause vision complications in patients.

The supplemental new drug application (sNDA) is based on results from a phase 3 trial that enrolled patients with metastatic hormone-sensitive prostate cancer (mHSPC).

ALE.P02 could provide further treatment options for advanced or metastatic anti–claudin-1+ squamous cancers.

The indication is for adults with previously treated unresectable or metastatic HER2+ (IHC+) biliary tract cancer (BTC) as detected by an FDA-approved test.

Bimekizumab-bkzx is the first and only approved medication that targets both interleukin (IL) 17F and IL-17A.

This marks the first and only therapy to receive this designation for Sjögren disease, which currently has no approved treatment.

Clinical trial analysis results studying VAD-44 in patients with hereditary hemorrhagic telangiectasia (HTT) will be presented at the 2024 American Society of Hematology Annual Meeting & Exposition.

GNSC-001 is a first-in-class gene therapy designed to offer long-term relief of musculoskeletal diseases such as osteoarthritis.

The disorder presents as severe lung disease, with lung transplantation as the only current therapeutic option for long-term survival.