(IN BRIEF) GSK announced that its B7-H3-targeted antibody-drug conjugate GSK5764227 (GSK’227) has been granted Orphan Drug Designation (ODD) by the European Medicines Agency (EMA) for the treatment of pulmonary neuroendocrine carcinoma, including small-cell lung cancer (SCLC). The designation is supported … Read the full press release
Posted in Business, Healthcare, Investment, Pharma & Biotech, Science, Technology, United Kingdom
Tagged ADC, antibody-drug conjugate, ARTEMIS-001, B7-H3, breakthrough therapy designation, cancer research, EMA, EMA Grants Orphan Drug Designation to GSK’s B7-H3 Antibody-Drug Conjugate for Pulmonary Neuroendocrine Carcinoma, FDA, GSK, GSK Strengthens Oncology Pipeline With EMA Orphan Drug Status for GSK’227 in Small-Cell Lung Cancer, GSK5764227, GSK’227, immuno-oncology, lung cancer, NEC, ODD, oncology, oncology pipeline, orphan drug designation, PRIME designation, pulmonary neuroendocrine carcinoma, relapsed or refractory, SCLC, Small cell lung cancer, solid tumours, targeted therapy
(IN BRIEF) Sanofi’s investigational therapy efdoralprin alfa met all primary and key secondary endpoints in the Phase 2 ElevAATe study for alpha-1 antitrypsin deficiency emphysema, demonstrating superior efficacy compared to standard plasma-derived therapy. The results highlight its potential as the … Read the full press release
Posted in France, Healthcare, Pharma & Biotech
Tagged AATD emphysema, alpha-1 antitrypsin deficiency, biotechnology, Christopher Corsico, clinical research, David Geffen School of Medicine, efdoralprin alfa, ElevAATe OLE, ElevAATe study, FDA Fast Track, functional AAT, Igor Barjaktarevic, orphan drug designation, phase 2 trial, protein replacement therapy, pulmonary medicine, rare disease, recombinant therapy, respiratory health, respiratory innovation, Sanofi, Sanofi’s Efdoralprin Alfa Shows Superior Results in Phase 2 AATD Emphysema Trial, SAR447537, UCLA
(IN BRIEF) The FDA has awarded orphan drug designation to Sanofi’s riliprubart for the treatment of antibody-mediated rejection in solid organ transplants, highlighting the therapy’s potential in an area lacking approved options. Riliprubart, a humanized IgG4 antibody targeting activated C1s … Read the full press release
Posted in Business, Financial, France, Government, Healthcare, Industrial, Investment, Management, Marketing, News, Pharma & Biotech, Security & Safety, Technology
Tagged Alyssa Johnsen, AMR, antibody-mediated rejection, C1s inhibition, chronic inflammatory demyelinating polyneuropathy, CIDP, ClinicalTrials.gov, complement pathway, FDA, Global Therapeutic Area Development Head, immunology, MOBILIZE, NCT05156710, NCT06290128, NCT06290141, orphan drug designation, Phase 2 study, riliprubart, Sanofi, SAR445088, solid organ transplantation, VITALIZE
(IN BRIEF) GSK’s marketing authorisation application for linerixibat, an investigational ileal bile acid transporter inhibitor, has been accepted for review by the European Medicines Agency. The submission is supported by GLISTEN Phase III trial data showing that linerixibat rapidly and … Read the full press release
Posted in Business, Healthcare, Pharma & Biotech, United Kingdom
Tagged alcohol-related liver disease, bile acid transporter, cholestatic pruritus, chronic hepatitis B, EASL Congress, EMA, European Medicines Agency, FDA, GLISTEN trial, GSK, IBAT inhibitor, itch-related sleep interference, Kaivan Khavandi, linerixibat, Marketing Authorisation Application, MASH, NCT04950127, orphan drug designation, PBC, Phase III, primary biliary cholangitis, UK regulatory review
(IN BRIEF) Rilzabrutinib, an oral Bruton’s tyrosine kinase (BTK) inhibitor, has received orphan drug designation from the U.S. FDA for sickle cell disease. This marks the fourth such designation for the drug, which has already been recognized for its potential … Read the full press release
Posted in Business, Financial, France, Government, Healthcare, Industrial, Investment, Management, News, Pharma & Biotech, Science, Technology
Tagged BTK inhibitor, fast track status, FDA, IgG4-related disease, immune thrombocytopenia, multi-immune modulation, orphan drug designation, preclinical data, rare diseases, rilzabrutinib, Sanofi, sickle cell disease, Tailored Covalency®, vaso-occlusive crises, warm autoimmune hemolytic anemia
(IN BRIEF) GSK has received approval from Japan’s Ministry of Health, Labour and Welfare (MHLW) for Blenrep combinations to treat adults with relapsed or refractory multiple myeloma. The approval follows positive results from the DREAMM-7 and DREAMM-8 phase III trials, … Read the full press release
Posted in Business, Environment, Financial, Government, Healthcare, Industrial, Investment, Management, News, Pharma & Biotech, Science, Security & Safety, Technology, United Kingdom
Tagged anti-BCMA, antibody-drug conjugate, Blenrep, bortezomib, chemotherapy, clinical efficacy, clinical trials, DREAMM-7, DREAMM-8, global markets, GSK, Japan approval, Multiple Myeloma, oncology, orphan drug designation, overall survival, partnership, PDUFA, pomalidomide, progression-free survival, regulatory approval, sustainability, treatment options
(IN BRIEF) Rilzabrutinib, an investigational oral BTK inhibitor, has received orphan drug designation from the FDA for treating warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD), two rare conditions with no approved treatments. This designation, aimed at addressing unmet … Read the full press release
Posted in Business, Financial, France, Government, Healthcare, Industrial, Investment, Management, News, Pharma & Biotech, Science, Security & Safety, Technology
Tagged BTK inhibitor, clinical trials, FDA, IgG4-RD, IgG4-related disease, immune thrombocytopenia, ITP, multi-immune modulation, orphan drug designation, phase 2a study, phase 2b study, rare diseases, regulatory review, rilzabrutinib, Sanofi, unmet medical need, wAIHA, warm autoimmune hemolytic anemia
(IN BRIEF) Sanofi’s Dupixent has been granted FDA priority review for the treatment of bullous pemphigoid (BP), a chronic skin disease. If approved, Dupixent would be the first targeted medicine for BP in the U.S., with the FDA’s decision expected … Read the full press release
Posted in Business, Financial, France, Healthcare, Industrial, Investment, Management, News, Pharma & Biotech, Science, Technology
Tagged bullous pemphigoid, chronic conditions, clinical trial, corticosteroid, disease remission, Dupixent, Dupixent sBLA, FDA approval, FDA priority review, orphan drug designation, regulatory news, Sanofi, skin disease, targeted treatment
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