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Ultragenyx Gets FDA Nod for Label Expansion of Crysvita

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Ultragenyx Pharmaceutical Inc. (RARE - Free Report) and partner Kyowa Kirin Co., Ltd. (Kyowa Kirin) announced that the FDA has approved a label expansion of Crysvita (burosumab) for the treatment of tumor-induced osteomalacia (TIO). The FDA approved the drugfor the treatment of fibroblast growth factor 23 (FGF23)-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older.

Sharesof Ultragenyx have surged 69.7% year to date compared with the industry’s growth of 9.2%.


Crysvita is an antibody targeting fibroblast growth factor 23 (FGF23), approved in the United States for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients. It is approved in the EU for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older, and adolescents with growing skeletons.TIO is the second FDA-approved indication for Crysvita.

The FDA approval of the drugmarks the first treatment option that addresses the cause of severe hypophosphatemia and osteomalacia resulting from these rare tumors.

The FDA approval of Crysvita for TIO was based on data from two, single-arm, phase II studies — a 144-week study in 14 adult patients conducted by Ultragenyx in the United States and an 88-week 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. 

We remind investors that the company submitted a supplemental biologics license application (sBLA) to the FDA on Dec 18, 2019, seeking approval for the label expansion of Crysvitafor the treatment of TIO. 

The approval of Crysvitato treat TIO will lead to increased sales for the company.

Ultragenyx has several products in its pipeline.

The company is developing a synthetic triglyceride-UX007 for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD). The FDA has accepted for review the NDA for the same and set an action date of Jul 31, 2020.

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. DTX401 is also an AAV8 gene-therapy candidate, currently being evaluated for the treatment of patients with glycogen storage disease type Ia (GSDIa). 

The company also has an agreement with Bayer (BAYRY - Free Report) to develop adeno-associated virus gene therapies, one of which isDTX 201 for the treatment of hemophilia A.


Zacks Rank and Stocks to Consider

Ultragenyx is a Zacks Rank #3 (Hold) stock.

Some better-ranked stocks in the biotech sector are Akcea Therapeutics Inc. (AKCA - Free Report) and Axcella Health Inc. (AXLA - Free Report) , both carrying a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Akcea’s loss per share estimates have narrowed from $2.07 to $1.37 for 2020 and from $2.46 to $1.85 for 2021 in the past 60 days.

Axcella’s loss per share estimates have narrowed from $1.14 to 79 cents for 2020 and from $3.30 to $3.03 for 2021 in the past 60 days.

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