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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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.
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.
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.