Ultragenyx Pharmaceutical Inc. RARE and partner Kyowa Kirin Co., Ltd. announced the receipt of the FDA approval for a label expansion of Crysvita (burosumab).
The label has been updated to include new clinical data demonstrating the superiority of treatment with Crysvita over oral phosphate and active vitamin D (conventional therapy) in pediatric patients with X-linked hypophosphatemia (XLH), improvement in stiffness, and maintenance of the efficacy of the drug in adult patients undergoing long-term treatment. The indication has also been expanded to include infants as young as six months.
The approval was supported by the 64-week efficacy and safety data from the randomized, active-controlled phase III study of Crysvita compared with oral phosphate and active vitamin D (conventional therapy) in 61 children with XLH. The study showed superior efficacy of the drug over conventional oral phosphate treatments.
Crysvita, an antibody targeting fibroblast growth factor 23 (FGF23), is now approved in the United States for the treatment of XLH in both adult and pediatric patients. The drug received European conditional marketing authorization in February 2018 for the treatment of XLH with radiographic evidence of bone disease in children aged one year or older, and adolescents with growing skeletons. The drug has been performing well and boosts sales for the company.
Ultragenyx also has some gene-therapy candidates in its pipeline. These include DTX301, which is an adeno-associated virus 8 (AAV8) being evaluated for the treatment of patients with Ornithine transcarbamylase (OTC) deficiency.
DTX401 is an AAV8 gene-therapy candidate being evaluated for the treatment of patients with glycogen storage disease type Ia (GSDIa).
All of Ultragenys’s current clinical product candidates are in-licensed from academic institutions or derived from partnerships with other pharmaceutical companies.
The company is developing DTX201 in its FVIII gene-therapy program for the treatment of hemophilia A in collaboration with Bayer BAYRY.
Ultragenyx exercised its option with REGENXBIO Inc. RGNX to develop a gene therapy for the treatment of patients with CDD, using REGENXBIO’s adeno-associated virus (AAV) vectors like AAV9.
Additionally, the company expanded its collaboration with Arcturus Therapeutics Holdings Inc. ARCT in June 2019 to discover and develop mRNA, DNA and siRNA therapeutics for up to 12 rare disease targets.
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