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Amirah Al Idrus
Europe follows FDA suit with approval spree this week
Friday, August 31, 2018
In a busy week at the European Commission, Novartis’ Kymriah, Gilead’s Yescarta, Jazz's Vyxeos, Alnylam's Onpattro and others got approvals.
U.K. cost watchdogs turn away rare disease med Crysvita
Friday, June 15, 2018
Patients in the U.K. won't have access to X-linked hypophosphatemia med Crysvita under a ruling by NICE.
Ultragenyx nabs FDA nod for $200K-per-year Crysvita
Wednesday, April 18, 2018
Ultragenyx is back with another FDA nod, this time for Crysvita to treat X-linked hypophosphatemia in patients one year and older.
Ultragenyx's ultraorphan med Mepsevii nabs FDA approval
Wednesday, November 15, 2017
Rare disease biotech Ultragenyx has its first FDA nod in Mepsevii, an enzyme therapy expected to carry a high price to treat a tiny group of patients.