Basel – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced results from the final analysis of the phase IIIb STASEY study, which confirm the favourable safety profile of Hemlibra® (emicizumab), consistent with the phase III HAVEN clinical programme.[1],[2],[3],[4] In the analysis, no new safety signals were identified with longer-term Hemlibra...
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Cross-study findings indicate significant clinical benefit of frontline fixed-duration ibrutinib plus venetoclax with improved likelihood of undetectable minimal residual disease and progression-free survival versus acalabrutinib plus venetoclax1 Phase 2 CAPTIVATE long-term follow-up data further supports sustained efficacy and safety profile of fixed-duration ibrutinib plus venetoclax treatment in patients receiving frontline...
New data from the phase II CITYSCAPE trial show encouraging results with Roche’s novel anti-TIGIT tiragolumab plus Tecentriq After 2.5 years median follow-up, pre-planned exploratory analyses in the PD-L1-high population show clinically meaningful results, with overall survival not yet reached and continued progression-free survival improvement for the combination compared with...
BOSTON, Mass. — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data across multiple studies demonstrating positive clinical and quality of life benefits of treatment with CFTR modulators and, in particular, ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor), which is approved in the United States and United Kingdom and is currently under review with health authorities in the EU, Canada, Australia, New Zealand and Switzerland. These data...
Philadelphia, PA – The new Kids First datasets advance our understanding of how genetics contribute to childhood cancers and congenital disorders, opening new doors for prevention and treatment. WHO: The Gabriella Miller Kids First Pediatric Research Program (Kids First), an initiative of the National Institutes of Health (NIH) WHAT: Kids...
Ingelheim/Germany — New data presented today at the Movement Disorder Society’s 13th International Congress of Parkinson’s Disease and Movement Disorders (MDS) further confirms high therapeutic benefit of Mirapexin®/Sifrol® (pramipexole) once-daily, extended release formulation (also referred to as prolonged release formulation). The trial, performed in patients with advanced Parkinson’s disease (PD),...
SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced data from the first study on the impact of caring for a child with the ultra-rare genetic disorder, aromatic L-Amino acid decarboxylase deficiency (AADC-d). Results show that caregivers spend almost every waking moment caring for a child with AADC-d,...
COPENHAGEN, Denmark — Ascendis Pharma A/S (Nasdaq: ASND) today announced new data showing improvements in growth and bone morphometry from Week 52 of its pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia. TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly providing...
SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that positive early results from an investigator-sponsored Phase 2 study of VOXZOGO® (vosoritide) in children with hypochondroplasia, will be presented at the 2024 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Toronto, Canada, March 12-16, 2024....
STOCKHOLM–– Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) today announced that its commercial partner Everest Medicines (HKEX 1952.HK) received approval from the Pharmaceutical Administration Bureau of the Macau Special Administrative Region, China. The approval for Nefecon is for the treatment of primary immunoglobulin A nephropathy (IgAN) in adults at risk...