Ascendis Pharma A/S’ Yuviwel (navepegritide) gained U.S. FDA accelerated approval Feb. 27 for children 2 and older with the rare genetic bone growth disorder achondroplasia, also known as dwarfism, winning the company a rare pediatric disease priority review voucher
Bridgebio Pharma Inc. kept the phase III wins coming, this time with positive top-line results from Propel 3, the global phase III pivotal study of oral infigratinib, designed to inhibit FGFR3 signaling in children with achondroplasia.
Ribomic’s umedaptanib pegol (RBM-007) looks to have some advantages compared to competitors in the achondroplasia space, and the company plans to progress the oligonucleotide-based aptamer that targets anti fibroblast growth factor 2 to phase III trials, Ribomic’s business development head, Kihei Yamashita, told BioWorld.
Ribomic’s umedaptanib pegol (RBM-007) looks to have some advantages compared to competitors in the achondroplasia space, and the company plans to progress the oligonucleotide-based aptamer that targets anti fibroblast growth factor 2 to phase III trials, Ribomic’s business development head, Kihei Yamashita, told BioWorld.
Predictably, weekly vs. daily subcutaneous injections made a big difference in the achondroplasia space, where shares of Copenhagen, Denmark-based Ascendis Pharma A/S (NASDAQ:ASND) closed Sept. 16 at $139.57, up $20.35, or 17%, on favorable, pivotal top-line data with Transcon CNP (navepegritide), as competitor Biomarin Pharmaceutical Inc.’s stock (NASDAQ:BMRN) ended the day at $69.86, down $15.04, or 17.7%.
Cogent Biosciences Inc. has disclosed compounds acting as fibroblast growth factor receptor (FGFR) inhibitors reported to be useful for the treatment of achondroplasia, cancer, craniosynostosis, Alzheimer's disease, fibrosis, pulmonary fibrosis, systemic scleroderma (systemic sclerosis) and thanatophoric dysplasia, among others.
Bridgebio Pharma Inc.’s data from the phase II study with infigratinib in achondroplasia “swings the pendulum of debate sharply in favor” of the firm, competing with Biomarin Pharmaceutical Inc. in the space, Leerink analyst Mani Foroohar said in a report.
Novo Nordisk A/S has identified C-type natriuretic peptide (CNP) compounds reported to be useful for the treatment of achondroplasia and heart failure.
Palo Alto, Calif.-based Bridgebio Pharma Inc. will hand over development and sales of its rare bone growth disorder therapy, infigratinib, in Japan to Kyowa Kirin Co. Ltd. under its latest exclusive licensing deal.
Palo Alto, Calif.-based Bridgebio Pharma Inc. will hand over development and sales of its rare bone growth disorder therapy, infigratinib, in Japan to Kyowa Kirin Co. Ltd. under its latest exclusive licensing deal.
