Ultragenyx Pharma (RARE), GeneTx Report Investigational New Drug Application Active for GTX-102 in Patients with Angelman Syndrome

Ultragenyx Pharma (RARE) Preliminary 2019 Revenue and Provides 2020 Crysvita Revenue Guidance

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported preliminary unaudited 2019 revenue and cash and investments at year end 2019, and provided 2020 revenue guidance for Crysvita in Ultragenyx territories.

We passed the $100 million revenue threshold in 2019 based on the strong launches of Crysvita and Mepsevii. We aim to build on this success in 2020 with the potential approvals of UX007 in LC-FAOD and Crysvita in TIO, said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx.... More

Ultragenyx Pharma (RARE), Kyowa Kirin Report Submission of Supplemental Biologics License Application to FDA for Crysvita (burosumab)

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd., (TYO: 4151) today announced that they submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) on December 18, 2019, for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The companies expect to hear back from FDA on submission acceptance and review designation in February... More

Ultragenyx Pharma (RARE) PT Raised to $53 at Citi

Citi analyst Yigal Nochomovitz raised the price target on Ultragenyx Pharma (NASDAQ: RARE) to $53.00 (from $45.00) while maintaining a Neutral rating.... More