Neurogene, a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, announces that the European Medicines Agency (EMA) has granted orphan drug designation (ODD) to its adeno-associated virus vector (AAV) with engineered transgene encoding the human AGA gene for patients diagnosed with aspartylglucosaminuria.
Roche announced that the U.S. Food and Drug Administration (FDA) has approved Gavreto (pralsetinib) for the treatment of adult and paediatric patients 12 years of age and older with advanced or metastatic rearranged during transfection (RET)-mutant medullary thyroid cancer (MTC) who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).
Pfizer and BioNTech have secured temporary emergency use authorisation (EUA) from the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) for their Covid-19 mRNA vaccine called BNT162b2.
Zymeworks, a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, today announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for zanidatamab in patients with previously-treated HER2 gene-amplified biliary tract cancer (BTC).
Moderna announced that it is seeking an emergency use authorisation (EUA) from the US Food and Drug Administration (FDA) for its Covid-19 mRNA vaccine candidate.
Vertex Pharmaceuticals has secured approval from the European Commission (EC) for Symkevi (tezacaftor/ivacaftor) plus Kalydeco (ivacaftor) for the treatment of elder children with cystic fibrosis (CF) aged between six and 11 years.
Merck announced that the European Medicines Agency (EMA) has validated for review, the application for tepotinib for the treatment of adult patients with advanced non-small cell lung cancer (NSCLC) harboring mesenchymal-epithelial transition factor gene (MET) exon 14 (METex14) skipping alterations.
Astellas Pharma and FibroGen have announced the receipt of approval from the Japan’s Ministry of Health, Labour and Welfare (MHLW) for Evrenzo (roxadustat) to treat anaemia in adult patients suffering from chronic kidney disease (CKD) but not on dialysis.
Revance Therapeutics announced that the United States (U.S.) Food and Drug Administration (FDA) has deferred a decision on the Biologics License Application (BLA) for DaxibotulinumtoxinA for Injection, an investigational neuromodulator product for the treatment of moderate to severe glabellar (frown) lines.
ADC Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for loncastuximab tesirine (Lonca) for the treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and granted priority review status
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