CLINICAL ROLE -
FDA Grants Fast Track Designation to VLS-1488 For Patients With Platinum-Resistant HGSOC
Currently, VLS-1488 is being evaluated in a phase 1/2 trial (NCT05902988) in various advanced tumors, including high-grade serous ovarian cancer (HGSOC).
FDA Grants Priority Review to Trastuzumab Deruxtecan for HER2-low, HER2 Ultralow Breast Cancer
The designation was based on results from the DESTINY-Breast06 trial, evaluating fam-trastuzumab deruxtecan-nxki (Enhertu; AstraZeneca, Daiichi Sankyo) compared with chemotherapy.
FDA Grants Denifanstat Breakthrough Therapy Designation for Treatment of MASH
Positive trial results laid the groundwork for the designation.
Navenibart Receives FDA Orphan Drug Designation to Treat Hereditary Angioedema
Navenibart was developed to aid treatment of hereditary angioedema (HAE) to provide swift and sustained HAE attack prevention through administration every 3 to 6 months.
FDA Approves Ustekinumab-Aauz as Biosimilar for Stelara
Ustekinumab is a human monoclonal antibody targeting the cytokines interleukin (IL)-12 and IL-23, which play a rule in the inflammatory and immune responses.
FDA Grants Rare Pediatric Disease Designation to MDL-101 for Congenital Muscle Dystrophy
MDL-101 is a proposed novel precision medicine that targets the LAMA1 gene, causing LAMA2 congenital muscular dystrophy type 1a.
FDA Approves Selpercatinib For Pediatric and Adult Patients With RET-Mutated MTC
The indication is for adult and pediatric patients aged 2 years and older with advanced or metastatic RET-mutated medullary thyroid cancer (MTC) who require systemic therapy.
FDA Approves Dupilumab as Add-On Maintenance Therapy For Adults With COPD
Dupilumab is the first biologic medicine for patients with chronic obstructive pulmonary disease to be approved in the US.
FDA Approves Cobenfy, Previously KarXT, for Treatment of Schizophrenia
Xanomeline and trospium chloride is the first in a new class, offering a new approach with selectively targeting M1 and M4 receptors.
You Previously Qualified for a Small Dispenser DSCSA Exemption. Now What?
As welcome as this compliance extension may be for those who qualify, it is critical they understand both their current and future regulatory obligations.
Levacetylleucine Receives FDA Approval as Stand-Alone Therapy for Niemann-Pick Disease Type C
The approval marks the only FDA-approved stand-alone therapy for the treatment of Niemann-Pick disease type C (NPC).
FDA Approves Osimertinib for Unresectable EGFR-Mutated Lung Cancer
The decision is based on positive results from the phase 3 LAURA trial.
FDA Grants Rare Pediatric Disease Designation to EXG-34217 for Dyskeratosis Congenita
Treatment is typically tailored to the individuals, with HCT being the only curative treatment bone marrow failure, but long-term outcomes are generally poorer due to toxicities.
FDA Accepts sNDA of Roflumilast Foam for Scalp and Body Psoriasis
The FDA assigned a Prescription Drug User Fee Act action date of May 22, 2025.
FDA Accepts New Drug Application for Delgocitinib Cream to Treat Chronic Hand Eczema
If approved, delgocitinib cream would be the first US treatment indicated for moderate to severe chronic hand eczema.
FDA Grants Bimekizumab-Bkzx Approval for Treatment of Chronic Immune-Mediated Inflammatory Diseases
Approved with 3 new indications, bimekizumab-bkzx is the first and only interleukin-17 inhibitor approved for these diseases.
FDA Approves Isatuximab With Bortezomib, Lenalidomide, and Dexamethasone for NDMM
The decision expands treatment options for patients with transplant-ineligible newly diagnosed multiple myeloma (NDMM).
Arimoclomol Becomes First FDA Approved Treatment for Neimann-Pick Disease Type C
The rare disease can result in progressive neurological symptoms and organ complications.
FDA Approves Amivantamab-vmjw for NSCLC With EGFR Exon 19 Deletions or L858R Mutations
Amivatamab-vmjw is a fully-human bispecific antibody targeting EGFR and MET with immune cell-directed activity.
FDA Grants Fast Track Designation to ICT01 in Combination With Azacitidine and Venetoclax for Acute Myeloid Leukemia
The humanized anti-butyrophilin 3A monoclonal antibody selectively activates Vγ9Vδ2 T-cells.
FDA Approves Pembrolizumab Plus Pemetrexed and Platinum Chemotherapy for Malignant Pleural Mesothelioma
The approval is based on positive results from the phase 2/3 IND.227/KEYNOTE-483 trial.
FDA Approves Benralizumab for Eosinophilic Granulomatosis With Polyangiitis
Eosinophilic granulomatosis with polyangiitis is a rare and immune-mediate vasculitis that can damage multiple organs and be fatal if left untreated.
FDA Approves Ribociclib in Combination With an Aromatase Inhibitor for HR+/HER2- Early Breast Cancer
The treatment significantly reduced risk of occurrence by 25% compared with endocrine therapy alone.
FDA Grants MM-II Fast Track Designation for Treatment of Osteoarthritis Knee Pain
The non-opioid drug can induce healing of joint cartilage in patients with osteoarthritis.
FDA Approves Lebrikizumab for Treatment of Atopic Dermatitis
Lebrikizumab (Ebglyss; Eli and Lilly Company) is a monthly maintenance injection with proven efficacy in adults and children aged 12 to 18 years.
Atezolizumab and Hyaluronidase-tqjs Subcutaneous Formulation Receives FDA Approval to Treat Multiple Forms of Cancer
Subcutaneous administration of atezolizumab and hyaluronidase-tqjs had similar efficacy to intravenous administration.
FDA Approves Guselkumab for Treatment of Ulcerative Colitis
Guselkumab (Tremfya; Johnson & Johnson) is the first and only approved fully human and dual-acting monoclonal antibody blocking interleukin-23 and binding to CD64 receptors.
FDA Grants Breakthrough Therapy Designation to Plozasiran for Familial Chylomicronemia Syndrome
FCS is a severe, rare genetic disease in which patients have extremely high triglyceride levels, typically above 880 mg/dL.
FDA Grants Rare Pediatric Disease Designation to NS-050/NCNP-03 to Treat Muscle Wasting Disease
NS-050/NCNP-03 is being developed to aid individuals with confirmed gene mutations that are treatable with exon 50 skipping therapy.
FDA Approves Sparsentan, With Data Indicating Slowing of Kidney Function Decline in IgA Nephropathy
Full traditional approval follows accelerated approval of the medication last year.
