CLINICAL ROLE -
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.
FDA Accepts sNDA for Darolutamide With ADT for Patients With mHSPC
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).
Antibody Drug Conjugate Targeting CLDN1 Receives FDA Fast Track Designation
ALE.P02 could provide further treatment options for advanced or metastatic anti–claudin-1+ squamous cancers.
Intravenous Injections of Zanidatamab-Hrii Receives FDA Accelerated Approval for IHC+ BTC
The indication is for adults with previously treated unresectable or metastatic HER2+ (IHC+) biliary tract cancer (BTC) as detected by an FDA-approved test.
FDA Approves Bimekizumab-Bkzx as Treatment for Hidradenitis Suppurativa
Bimekizumab-bkzx is the first and only approved medication that targets both interleukin (IL) 17F and IL-17A.
FDA Grants Breakthrough Therapy Designation for Nipocalimab to Treat Sjögren Disease
This marks the first and only therapy to receive this designation for Sjögren disease, which currently has no approved treatment.
FDA Grants Fast Track Designation to VAD044 for Treatment of Adults With HHT
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.
FDA Grants Fast Track Designation to Potential Gene Therapy for Osteoarthritis of the Knee
GNSC-001 is a first-in-class gene therapy designed to offer long-term relief of musculoskeletal diseases such as osteoarthritis.
FDA Grants Rare Pediatric Disease Designation for Treatment of Neonatal SP-B Deficiency
The disorder presents as severe lung disease, with lung transplantation as the only current therapeutic option for long-term survival.
FDA Approves Revumenib for the Treatment of Relapsed or Refractory Acute Leukemia
The indication is for adult and pediatric patients 1 year and older with a lysine methyltransferase 2A gene translocation.
FDA Accepts sBLA Review for Dupilumab in Treatment of CSU in Adult and Pediatric Patients
The indication is for patients 12 and older with chronic spontaneous urticaria (CSU) whose disease is not adequately controlled with H1 antihistamine treatment.
FDA Grants New Nilotinib Formulation Approval to Treat Adults With Chronic Myeloid Leukemia
The tablets become the first and only nilotinib with no mealtime restrictions indicated for this population.
FDA Adds Delayed Gastric Emptying as Adverse Event on Semaglutide Label
The label includes postmarking reports showing rare instances of pulmonary aspiration for patients undergoing procedures that require general anesthesia or deep sedation.
FDA Approves Eladocagene Exuparvovec-Tneq for Treatment of AADC Deficiency
Eladocagene exuparvovec-tneq is the first FDA-approved gene therapy for individuals with aromatic L-amino acid decarboxylase deficiency.
FDA Grants Rare Pediatric Disease Designation to Elraglusib for Ewing Sarcoma
The designation follows 2 previous orphan drug designations for pancreatic cancer and soft tissue sarcoma.
Bispecific Antibody Receives FDA Orphan Drug Designation for the Treatment of Multiple Myeloma
LBL-034 could be best in class in treating individuals with multiple myeloma.
