16:32 , Apr 20, 2018 |  BC Week In Review  |  Clinical News

Ultragenyx gains rare disease approval, voucher

FDA approved Crysvita burosumab-twza (KRN23, UX023) from Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) and partner Kyowa Hakko Kirin Co. Ltd. (Tokyo:4151) to treat X-linked hypophosphatemia in patients aged one year and older. FDA said Crysvita is the...
19:57 , Apr 17, 2018 |  BC Extra  |  Company News

Ultragenyx gains rare disease approval, voucher

FDA approved Crysvita burosumab-twza (KRN23, UX023) from Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) and partner Kyowa Hakko Kirin Co. Ltd. (Tokyo:4151) to treat X-linked hypophosphatemia in patients aged one year and older. FDA said Crysvita is the...
19:26 , Dec 22, 2017 |  BC Week In Review  |  Company News

Novartis buys Priority Review voucher from Ultragenyx

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said it will sell its rare pediatric disease Priority Review voucher to Novartis AG (NYSE:NVS; SIX:NOVN) for $130 million. Ultragenyx received the voucher in November, when FDA approved Mepsevii vestronidase alfa-vjbk...
19:52 , Dec 18, 2017 |  BC Extra  |  Company News

Novartis buying Ultragenyx’s Priority Review voucher

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said it will sell its rare pediatric disease Priority Review voucher to Novartis AG (NYSE:NVS; SIX:NOVN) for $130 million. Ultragenyx received the voucher in November, when FDA approved Mepsevii vestronidase alfa-vjbk...
18:13 , Dec 8, 2017 |  BioCentury  |  Product Development

Making of Mepsevii

It may take a few years for Ultragenyx Pharmaceutical Inc. to recoup its investment in Mepsevii vestronidase alfa-vjbk, but the company expects to continue to reliably churn out treatments for ultra-rare diseases alongside a portfolio...
03:49 , Nov 17, 2017 |  BC Week In Review  |  Clinical News

FDA approves Ultragenyx's Mepsevii for MPS VII

FDA approved Mepsevii vestronidase alfa-vjbk (rhGUS, UX003) from Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) to treat mucopolysaccharidosis VII (MPS VII; Sly syndrome). Mepsevii is the first therapy approved for the indication. Ultragenyx said Mepsevii will be available to...
21:12 , Nov 15, 2017 |  BC Extra  |  Company News

FDA approves Ultragenyx's Mepsevii for MPS VII

FDA approved Mepsevii vestronidase alfa-vjbk (rhGUS, UX003) from Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) to treat mucopolysaccharidosis VII (MPS VII; Sly syndrome). Mepsevii is the first therapy approved for the indication. Ultragenyx said Mepsevii will be available to...
23:10 , Oct 6, 2017 |  BioCentury  |  Finance

Coasting on catalysts

While buysiders do not expect any sector-moving milestones in the wake of a catalyst-heavy third quarter, they will have at least 21 Phase III milestones and 23 PDUFA dates on their radar. But the biggest...
20:51 , Sep 29, 2017 |  BioCentury  |  Regulation

Beyond compassion

Patient advocates, with support from FDA, are honing new arguments to persuade drug companies to routinely provide pre-approval access to investigational drugs. Rather than appealing to companies’ compassion, or threatening to blacken their reputations through...
20:10 , Sep 22, 2017 |  BC Week In Review  |  Company News

Ultragenyx counters RegenxBio with bid for Dimension

On Sept. 18, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) offered to acquire gene therapy play Dimension Therapeutics Inc. (NASDAQ:DMTX) for $5.50 per share in cash, or about $138 million. In August, RegenxBio Inc. (NASDAQ:RGNX) agreed to acquire...