Scrip is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By

UsernamePublicRestriction

Ultragenyx Gets Commercial Feet Wet With Mepsevii Launch In Sly Syndrome

Executive Summary

Commercial opportunity for Mepsevii is small, but launch paves way for company's second product burosumab, following on its heels.

You may also be interested in...



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.

Topics

Related Companies

Latest Headlines
See All
UsernamePublicRestriction

Register

SC099898

Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

Thank you for submitting your question. We will respond to you within 2 business days. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts

Cancel