Ultragenyx Gets Commercial Feet Wet With Mepsevii Launch In Sly Syndrome
Commercial opportunity for Mepsevii is small, but launch paves way for company's second product burosumab, following on its heels.
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Finance Watch: Another Mega-Round As Moderna Bucks Biopharma's IPO Trend
Moderna raised $500m to fund its growing pipeline, choosing once again to stay private rather than pursue an IPO. Also, AvroBio closed a $60m Series B round to advance its gene therapies for lysosomal storage disorders, Seattle Genetics led recent public company financings, and Vical is restructuring.
Novel Biologics Will Get Suffixes (Or At Least One Did)
US FDA's approval of Ultragenyx's Mepsevii (vestronidase alfa-vjbk) marks first novel biologic with a suffix, but 10 months after release of the naming guidance how – and even how widely – FDA will implement the policy remains uncertain.
Will Ultragenyx/Kyowa Hypophosphatemia Data Support Uptake?
Ultragenyx and partner Kyowa Hakko Kirin said burosumab (KRN23) met the primary endpoint regarding phosphate levels in a Phase III study that enrolled adult X-linked hypophosphatemia patients, but small effects on two out of three secondary endpoints could impact the product's use.