Kyowa Kirin's Crysvita conditionally OK'd in Europe of rare musculoskeletal disorder in children [Seeking Alpha]
Ultragenyx Pharmaceutical Inc. (RARE)
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm
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Source: Seeking Alpha
Kyowa Kirin's Crysvita conditionally OK'd in Europe of rare musculoskeletal disorder in childrenKyowa Hakko Kirin Co. and collaboration partner Ultragenyx Pharmaceutical (RARE-0.5%)announce that the European Commission has conditionally approved Crysvita (burosumab) for the treatment of children and adolescents withX-linked hypophosphatemia (XLH), a rare progressive musculoskeletal disorder also called X-linked vitamin D-resistant rickets. The disease leads to diminished height and deformity in the lower limbs.The aim ofconditional approval is early access to patients with an unmet medical need. Additional (late-stage) data are still required for full approval.Burosumab is a fully human recombinant monoclonla IgG1 antibody that binds to (inhibits) the phosphaturic hormone fibroblast growth factor 23 (FGF23), a hormone that reduces blood levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney.Ultragenyx's U.S. marketing
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News
- Ultragenyx (RARE) Meets Efficacy Goal in Neuro Disease Study [Yahoo! Finance]Yahoo! Finance
- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) had its "neutral" rating re-affirmed by analysts at Wedbush. They now have a $48.00 price target on the stock.MarketBeat
- Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102 [Yahoo! Finance]Yahoo! Finance
- Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102GlobeNewswire
- Ultragenyx Announces Update to Conference Call Timing to Discuss New Data from GTX-102 in Patients with Angelman Syndrome [Yahoo! Finance]Yahoo! Finance
RARE
Earnings
- 2/15/24 - Beat
RARE
Sec Filings
- 4/15/24 - Form 8-K
- 3/12/24 - Form 4
- 3/11/24 - Form 144
- RARE's page on the SEC website