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Ultragenyx's Dojolvi Gets FDA Nod to Treat Rare Disease

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Ultragenyx Pharmaceutical Inc. (RARE - Free Report) announced that the FDA has approved UX007 (triheptanoin) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). The drug will be marketed under the brand name,Dojolvi. The approval date comes a month ahead of the action date of Jul 31.

Ultragenyx expects Dojolvi to be available to patients in the next 30 days. 

Long-chain fatty acid oxidation disorders are a group of rare conditions that prevent the body from breaking down certain fats into energy, leaving patientswith low blood sugar, muscle ruptures, muscle weakness and heart disease.

The approval of the drug will lead to increased sales for the company.

Shares of Ultragenyx have surged 83.1% year to date compared with the industry’s growth of 12.5%.


This is the second approval in June 2020 that the company received fromthe FDA for a rare disease, which had no approved therapy. In June, the FDA also approved the company’s Crysvita to treat tumor-induced osteomalacia, which is a rare disease that causes weakened or soft bones.

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). 

Further, the company 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 Emergent Biosolutions Inc. (EBS - Free Report) and Ligand Pharmaceuticals Inc. (LGND - Free Report) , both carrying a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Emergent’s earnings per share estimates have increased from $3.43 to $4.03 for 2020 and from $3.54 to $3.73 for 2021 in the past 60 days.

Ligand Pharmaceuticals’ earnings per share estimates have increased from $3.45 to $3.70 for 2020 and from $4.00 to $4.40 for 2021 in the past 60 days.

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