CLINICAL ROLE -
FDA Approves Crinecerfont for Adult, Pediatric Patients with Congenital Adrenal Hyperplasia
The decision is based on positive results from the CAHtalyst trials, which both met their primary and secondary end points.
Sacituzumab Govitecan-hziy Receives FDA Breakthrough Therapy Designation for Extensive-Stage Small Cell Lung Cancer Treatment
Abstract data presented in September demonstrate that the drug elicited strong antitumor activity in the second line in both platinum-sensitive and -resistant patients.
FDA Grants Breakthrough Therapy Designation to Tolebrutinib for Non-Relapsing Secondary Progressive Multiple Sclerosis
Based on positive results from the HERCULES trial, the new designation will allow for more thorough regulatory review and development of the multiple sclerosis treatment.
Nemolizumab Granted FDA Approval For Moderate-to-Severe Atopic Dermatitis
Nemolizumab exclusively targets IL-31 receptor alpha, inhibiting the signaling of IL-31, which drives disease mechanism in atopic dermatitis.
FDA Approves Cosibelimab-Ipdl for Adult Patients with mCSCC or laCSCC
The indication is for adults with metastatic cutaneous squamous cell carcinoma (mCSCC) or locally advanced CSCC (laCSCC) who are not candidates for curative surgery or radiation.
FDA Approves Tapinarof Cream 1% for Treatment of Atopic Dermatitis in Adults, Children 2 and Older
The approval builds on previous indications for tapinarof, an aryl hydrocarbon receptor agonist, for the topical treatment of atopic dermatitis.
FDA Grants Breakthrough Therapy Designation to Tobevibart With Elebsiran for Treatment of Chronic Hepatitis Delta
The treatments are currently undergoing evaluation in the SOLSTICE trial (NCT05461170).
FDA Expands Indication for Impella Heart Pumps to Include Pediatric Patients
The pumps can now be used to treat pediatric patients who have symptomatic acute decompensated heart failure and cardiogenic shock.
FDA Grants Fast Track Designation to IMM-1-104 for Advanced Melanoma
Currently, the treatment is undergoing evaluation in a phase 2a trial.
FDA Reports Serious Liver Injury in Patients Without Cirrhosis Using Obeticholic Acid to Treat PBC
This update follows May 2021 guidelines which recommended patients with primary biliary cholangitis (PBC) with advanced cirrhosis discontinue obeticholic acid.
FDA Grants Fast Track Designations to 2 Combination Influenza and COVID-19 Vaccine Candidates
The combination vaccine candidates will be evaluated in 2 separate phase 1/2 clinical trials.
FDA Grants PLT012 Orphan Drug Designation for Liver, Intrahepatic Bile Duct Cancer
PLT012 demonstrates its potential against multiple tumors with unmet medical needs.
IV Form of NAC Receives FDA sNDA to Prevent, Lessen Liver Injury After Ingesting Toxic Quantities of Acetaminophen
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.
FDA Accepts NDA for Paltusotine to Treat Adult Patients With Acromegaly
If approved, the treatment will be the first and only once-daily, oral selective somatostatin receptor type 2 nonpeptide agonist available for acromegaly.
Experts Voice Concerns, Cautious Optimism With Kennedy Potentially Leading the HHS
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.
FDA Grants Priory Review for Human Monoclonal Antibody Drug to Treat Muscle-Invasive Bladder Cancer
Durvalumab could offer further treatment for individuals with muscle-invasive bladder cancer.
FDA Grants Orphan Drug Designation to Novel Gene Therapy for Fabry Disease
Fabry disease is a rare lysosomal disorder that causes excessive deposition of lipids in tissues.
Zenocutuzumab-zbco Granted Accelerated Approval for Patients With NSCLC, Pancreatic Ductal Adenocarcinoma
Zenocutuzumab-zbco is a bispecific antibody that targets and binds to HER2 and HER3.
FDA Approves Durvalumab for Treatment of Adults Patients With LS-SCLC
The indication is for adults with limited-stage small cell lung cancer (LS-SCLC) whose disease has not progressed after platinum-based chemotherapy and radiation therapy.
FDA Grants Breakthrough Therapy Designation to Sac-TMT for Previously Treated NSCLC
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.
FDA Blocks Flavored E-Cigarettes, Sparking Legal Fight Over Risks to Minors
E-cigarette manufacturers claim the products aid in smoking cessation for adults without the risk of adolescent usage.
FDA Receives 2 Supplemental Biologics License Applications for Guselkumab for Pediatric Indications
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.
FDA Approves Ustekinumab-kfce as Sixth Biosimilar to Stelara
The biosimilar is approved for the treatment of Crohn disease, ulcerative colitis, plaque psoriasis, and psoriatic arthritis.
FDA Approves Landiolol For Treatment of Supraventricular Tachycardia
The approval is based on clinical studies that demonstrate the management of heart rate with minimal reductions to blood pressure.
FDA Is Evaluating the Prospect of Removing Phenylephrine From OTC Shelves
Oral phenylephrine is a common ingredient in OTC products, but its clinical efficacy is questionable. The FDA is considering removing it from the market.
FDA Approves Oral Liquid Solution of Imatinib for Certain Forms of Leukemia and Other Cancers
This is the first oral liquid form of imatinib (Imkeldi; Shorla Oncology) to be approved to treat cancers.
Acoramidis Received FDA Approval for Treatment of Transthyretin Amyloid Cardiomyopathy
Acoramidis is a novel, highly potent transthyretin stabilizer.
Arrowhead Pharmaceuticals Submits FDA Application for Plozasiran in Familial Chylomicronemia Syndrome
Familial chylomicronemia syndrome (FCS) currently has no FDA-approved treatment and is associated with an increased risk of acute pancreatitis.
FDA Grants Orphan Drug Designation to RAG-21 for Treatment of ALS
If approved, RAG-21 can offer improved outcomes in patients with amyotrophic lateral sclerosis (ALS), particularly those with aggressive subtypes.
FDA Grants Rare Pediatric Disease, Fast Track Designations to Gildeuretinol for Stargardt Disease Treatment
As a rare and serious pediatric disorder, Stargardt disease can cause vision complications in patients.
