CLINICAL ROLE -
FDA Accepts Application for 5-in-1 Meningococcal Vaccine Candidate
The 5-in-1 meningococcal ABCWY vaccine candidate has an assigned Prescription Drug User Fee Act action date of February 14, 2025.
FDA Grants Orphan Drug Designation for the Active Ingredient in MVD Vaccine
Although the vaccine has not yet been tested in humans, it demonstrated 100% efficacy in protecting primates who were injected with human Marburg virus disease.
FDA Grants Fast Track Designation for LYT-200 to Treat Head and Neck Cancers
Treatment with LYT-200 is currently being assessed in a phase 1/2 adaptive design trial in advanced/metastatic solid tumors and in a phase 1b clinical trial.
FDA Grants Orphan Drug Designation for CAN-2409 to Treat Pancreatic Cancer
The designation was granted based on overall survival data from an ongoing randomized phase 2 clinical trial.
FDA Updates Approval of Antiseizure Medication Cenobamate Allowing for Alternative Administration Methods
In addition to oral administration, patients with partial-onset seizures can now take the antiseizure medication via a nasogastric tube if the tablet is crushed and mixed with water.
FDA Approves Benralizumab to Treat Pediatric Patients Aged 6 to 11 Years With Severe Asthma
Benralizumab was previously approved by the FDA in 2017 as an add-on maintenance therapy for patients 12 years and older.
FDA Approves Cilta-Cel for Treatment of Relapsed, Refractory Multiple Myeloma
The indication is for adult patients with multiple myeloma who are refractory to lenalidomide and have previously received at least 1 line of therapy.
FDA Approves Dolutegravir/Lamivudine for Treatment of Adolescents With HIV-1 Infection
The treatment is indicated for adolescents at least 12 years of age who weigh at least 25 kg without a previous treatment history of antiretroviral therapies.
FDA Approves Fam-Trastuzumab Deruxtecan-nxki for Unresectable or Metastatic HER2-Positive Solid Tumors
This is the fifth indication for fam-trastuzumab deruxtecan-nxki (Enhertu; Daiichi Sankyo and AstraZeneca) approved by the FDA.
FDA Approves Idecabtagene Vicleucel for Multiple Myeloma After 2 or More Lines of Therapy
The approval expands the prior indication of idecabtagene vicleucel (Abecma; Bristol Myers Squibb), which will make the drug available to patients in earlier lines.
FDA Accepts sBLA for Bimekizumab-bkzx in Hidradenitis Suppurativa Treatment
Bimekizumab-bkzx (Bimzelx; Union Chimique Belge) is a humanized monoclonal immunoglobulin G1 antibody that inhibits interleukin (IL)-17A and IL-17F.
FDA Approves Zevtera as Antibiotic to Treat 3 New Indications
New indications include Staphylococcus aureus bloodstream (SAB) infections, acute bacterial skin and skin structure infections, and community-acquired bacterial pneumonia.
FDA Accepts BLA for Dato-DXd to Treat Unresectable, Metastatic HR-Positive, HER2-Negative Breast Cancer
The decision follows phase 3 trial results that demonstrated improved progression-free survival and confirmed the safety of Dato-DXd in patients with breast cancer.
FDA Approves Danicopan With C5 Inhibitor Therapy to Treat EVH in Adults With Paroxysmal Nocturnal Hemoglobinuria
Positive phase 3 trial results showed that danicopan was more effective than placebo when treating extravascular hemolysis in paroxysmal nocturnal hemoglobinuria.
FDA Grants ODD to AP303 for Treatment of Patients With Autosomal Dominant Polycystic Kidney Disease
The ODD comes after AP303 presented meaningful improvements in renal survival in an ADPKD and the completion of the first study that evaluated healthy human participants.
FDA Approves Expanded Indication for Tenofovir Alafenamide as Hepatitis B Treatment
The new indication includes pediatric patients aged 6 years and older who weigh at least 25 kg and have compensated liver disease.
FDA Approves Vadadustat for Oral Treatment of Anemia Due to Chronic Kidney Disease
Vadadustat (Vafseo; Akebia Therapeutics Inc) is indicated for individuals with chronic kidney disease who have been receiving dialysis for at least 3 months.
FDA Issues Letters to 6 Companies for Unapproved, Misbranded OTC Analgesic Products
The agency warns consumers against using these products, which are marketed for topical use to relieve pain associated with cosmetic procedures.
FDA Approves Sotatercept-csrk for Pulmonary Arterial Hypertension
Sotatercept-csrk is the first approved activin signaling inhibitor therapy for pulmonary arterial hypertension, which represents a new class of therapy.
Biosense Webster Announces FDA Submission for Approval of the Varipulse Platform
All 20 participants in a 12-month follow-up study attained acute success from ablation procedures.
FDA Approves Cobas Malaria to Help Reduce Risks Infections From Transfused Blood Products
The test scans whole blood samples from individual human donors for the 5 parasite species that can cause malaria in humans and detects Plasmodium RNA and DNA.
FDA Grants Fast Track Designation to Nipocalimab for Fetal Neonatal Alloimmune Thrombocytopenia
In December 2023, nipocalimab (Johnson & Johnson) was granted orphan drug designation for fetal neonatal alloimmune thrombocytopenia.
FDA Grants Orphan Drug Designation to Felzartamab for Kidney Transplant Recipients
Previously, felzartamab received orphan drug designation and breakthrough therapy designation for the treatment of primary membranous nephropathy.
FDA Grants ODD to Cevidoplenib for the Treatment of Immune Thrombocytopenia
The orphan drug designation (ODD) comes after positive results from a phase 2 trial that showed cevidoplenib improved platelet counts by 63.6% and 40.9% in 2 different dose groups.
FDA Approves Macitentan, Tadalafil Combination Tablet for PAH
The combination tablet (Opsynvi; Johnson & Johnson) can also be used for individuals with pulmonary arterial hypertension who are being treated with stable doses of macitentan and tadalafil as separate tables.
FDA Approves Ravulizumab-cwvz for Neuromyelitis Optica Spectrum Disorder
Zero relapses were reported among individuals that received ravulizumab-cwvz over the 73 weeks of treatment.
FDA Grants Pemivibart Emergency Use Authorization for PrEP of COVID-19 in Immunocompromised Patients
The indication is for adults and adolescents with moderate to severe immune compromise due to medical conditions or immunosuppressive medications and treatments.
FDA Grants ODD to Utidelone Capsules For Treatment of Gastric Cancer
The orphan drug designation (ODD) is a result of positive findings from a phase 2 clinical trial which demonstrated a 100% clinical benefit rate in patients.
FDA Grants Fast Track Designation to PT886 for the Treatment of Pancreatic Cancer
PT886 (Phanes Therapeutics) received fast track designation 2 years after receiving orphan drug designation by the FDA.
FDA Expands Label for Bempedoic Acid to Reduce Cardiovascular Risk, With or Without Statins
Bempedoic acid (Nexletol; Esperion) and bempedoic acid and ezetimibe (Nexlizet; Esperion) are the only LDL-C lowering non-statin drugs indicated for primary prevention patients.
