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Ultragenyx's (RARE) UX007 Gets Fast Track Status by FDA

Zacks Equity Research

Ultragenyx Pharmaceutical Inc.RARE announced that the FDA has granted Fast Track designation and Rare Pediatric Disease designation to UX007 for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD). Patients with this disorder are unable to properly break down fatty acids into energy, leading to serious liver, muscle and heart issues.

Shares of the company have increased 24.4% in the past year against the industry’s decline of 11.6%.

The FDA grants Fast Track designation to help the development and a faster review of drugs, which treat serious and unmet medical conditions. With this designation, the drug is expected to be granted priority review, once the company files a new drug application (“NDA”).

Additionally, the rare pediatric disease designation granted to UX007provides Ultragenyx withthe opportunity to obtain a Pediatric Disease Priority Review Voucher from the FDA, after a qualifying new drug application (NDA) or biologics license application (BLA) is approved for the treatment of any rare pediatric disease. The company may use the voucher to obtain priority review for an NDA or BLA submitted later.

Ultragenyx is on track to submit the NDA for UX007 in mid-2019. The submission will include data from a long-term efficacy and safety extension study in 75 patients and the phase II study of UX007 in 29 patients. The submission will also provide a retrospective medical record review of 20 original compassionate use patients, data from 70 patients treated through expanded access, and a randomized controlled investigator-sponsored study involving 32 patients showing the effect of the candidate on cardiac function.

Ultragenyx has a number of products in its pipeline. The company is also looking to expand the label of Crysvita. The drug is being developed for the treatment of tumor-induced osteomalacia, (TIO). The company also has some gene therapy candidates like DTX301, which is an adeno-associated virus 8 (AAV8) being evaluated for the treatment of patients with Ornithine transcarbamylase (OTC) deficiency. DTX401 is another AAV8 gene-therapy candidate being evaluated for the treatment of patients with glycogen storage disease type Ia (GSDIa).

Ultragenyx is developing DTX201, its FVIII gene-therapy program for the treatment of hemophilia A, in collaboration with Bayer BAYRY.

A potential approval of these candidates will drive the company’s revenues.


Ultragenyx Pharmaceutical Inc. Price


Ultragenyx Pharmaceutical Inc. Price | Ultragenyx Pharmaceutical Inc. Quote

Zacks Rank & Stocks to Consider

Ultragenyx currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks from the same space are Ani Pharmaceuticals Inc.  ANIP and Celsion Corporation CLSN, each carrying a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Ani Pharmaceuticals’ earnings per share estimates have increased from $5.90 to $5.97 for 2019 and from $6.58 to $6.62 for 2020 over the past 60 days. The company delivered a positive earnings surprise in three of the trailing four quarters at an average of 2.30%.

Celsion’s loss per share estimates have narrowed from 94 cents to 78 cents for both 2019 and 2020 over the past 60 days. The company delivered a positive earnings surprise in one of the trailing four quarters at an average of 33.45%.

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