CLINICAL ROLE -
Facing Industry Disruptions, FDA Grants Temporary Exemption From DSCSA Requirements For Trading Partners
The exemption allows more time for partners to adhere to enhanced distribution security requirements in the Food, Drug, & Cosmetics Act and prevents possible supply chain disruptions.
FDA Approves Inavolisib With Palbociclib and Fulvestrant for Breast Cancer
The FDA also approved the FoundationOne Liquid CDx assay as a companion diagnostic device to identify patients who would benefit from the treatment.
FDA Grants Breakthrough Therapy Designation for Volixibat in Cholestatic Pruritus From Primary Biliary Cholangitis
The regulatory action is based on a positive preliminary analysis of the VANTAGE trial.
FDA Grants Marketing Authorization for Rapid COVID-19/Influenza Antigen Test
Healgen Rapid Check COVID-19/Flu A&B Antigen Test is the first OTC test to detect influenza to be granted a marketing authorization outside of Emergency Use Authorizations.
FDA Grants Survodutide Breakthrough Therapy Designation for Treatment of Adults With MASH
Survodutide is a glucagon/glucagon-like peptide-1 (GLP-1) receptor dual agonist that activates the glucagon and GLP-1 receptors to better control metabolic function.
FDA Grants Breakthrough Therapy Designation to Setrusumab for Osteogenesis Imperfecta
Setrusumab could offer a rapid and clinically meaningful decrease in fracture rate in individuals with osteogenesis imperfecta.
FDA Approves Nivolumab for the Treatment of Adult Patients With Resectable NSCLC
Nivolumab is the only PD-1 inhibitor to show statistically significant and clinically meaningful benefits in non-small cell lung cancer compared with chemotherapy.
FDA Grants Orphan Drug Designation to LQT-1213 to Treat Patients With Long QT Syndrome
The treatment aims to reduce prolonged QTc interval in patients with long QT syndrome.
Cologuard Plus Receives FDA Approval For Non-Invasive Colorectal Cancer Screening
The new screening test provides an easier, convenient option for patients seeking to screen themselves for colorectal cancer.
FDA Grants Orphan Drug Designation to PPL-002 for the Treatment of Danon Disease
Currently, Danon disease has a poor prognosis and does not have any pharmaceutical therapeutics for treatment or management.
FDA Grants IND for RespiRx Inhalable Nicotine Replacement Therapy
In a phase 1 trial, the nicotine replacement therapy (NRT) demonstrated a higher mean maximum plasma concentration was achieved faster than with an existing inhaled NRT.
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.
