Aug. 27 Quick Takes: BioMarin gains first worldwide approval for achondroplasia therapy

The European Commission approved Voxzogo vosoritide from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) to treat children with achondroplasia, marking the first approval worldwide for the CNP analog that inhibits FGFR3 signaling. BioMarin said the price in France and Germany would be about $300,000 per patient annually. The product remains under FDA review, with a Nov. 20 PDUFA date.

Roche voluntarily withdrew its accelerated approval for blockbuster Tecentriq atezolizumab to treat metastatic triple negative breast cancer. The PD-L1 inhibitor gained the indication in combination with Abraxane nab-paclitaxel in March 2019 based on the progression-free survival benefit shown in the Phase III IMpassion130 study, but a readout in the confirmatory Phase III IMpassion131 study showed no PFS benefit, missing the primary endpoint. Calls have arisen for the FDA to revise the accelerated approval paradigm. ...