Ultragenyx Pharmaceutical Inc. (RARE - Free Report) and partner Kyowa Kirin Co., Ltd., announced plans to submit a supplemental Biologics License Application (sBLA) to the FDA for the label expansion of Crysvita (burosumab). The sBLA will be submitted for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The company decided to to submit the sBLA after completing a pre-sBLA meeting with the FDA and an agreement on the filing package. The submission of the sBLA is planned for the first half of 2020.
The sBLA will be supported by data from two single-arm phase II studies — a 144-week phase II study in 14 adult patients conducted by Ultragenyx in the United States and an 88-week phase II study in 13 adult patients conducted by Kyowa Kirin in Japan and South Korea. In both studies, Crysvita was associated with increases in serum phosphorus and serum 1,25-dihydroxyvitamin D levels. Increased phosphate levels led to improvements in osteomalacia, mobility, and vitality.
Shares of the company have inched up 0.9% year to date against the industry’s decline of 1.9%.
Crysvita, an antibody targeting fibroblast growth factor 23 (FGF23), is approved in the United States for the treatment of X-linked hypophosphatemia (XLH) in 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 one year of age or older, and adolescents with growing skeletons.
The company 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. It reported data from the first two dose cohorts of DTX301 gene therapy in the same indication. DTX301 phase I/II data from the third dose cohort are expected in the third quarter of 2019.
DTX401 is an AAV8 gene-therapy candidate being evaluated for the treatment of patients with glycogen storage disease type Ia (GSDIa).
The company is developing DTX201 in its FVIII gene-therapy program for the treatment of hemophilia A in collaboration with Bayer (BAYRY). The investigational new drug (IND) application was accepted by the FDA in the second quarter of 2018.
Zacks Rank & Stocks to Consider
Ultragenyx is currently a Zacks Rank #3 (Hold) stock.
Some better-ranked stocks in the biotech sector are Amgen Inc. (AMGN - Free Report) , Acorda Therapeutics Inc. (ACOR - Free Report) and Celcuity Inc. (CELC - Free Report) . All of these sport a Zacks Rank #1 (Strong Buy), at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Amgen’s earnings per share estimates have increased from $13.90 to $14.30 for 2019, and from $14.78 to $15.42 for 2020, in the past 60 days. The company delivered a positive earnings surprise in the trailing four quarters, the average beat being 6.72%.
Acorda’s loss per share estimates for the current year narrowed from $3.59 to $2.74 over the past 60 days. The company recorded a positive earnings surprise in the preceding four quarters, the average beat being 69.68%.
Celcuity’s loss per share estimates narrowed from 81 cents to 70 cents for 2019 and from 96 cents to 71 cents for 2020, in the past 60 days. The company delivered a positive earnings surprise in three of the trailing four quarters, the average beat being 25.76%.
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