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Ultragenyx (RARE) Gets Orphan Drug Tag for Wilson Disease Drug

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Ultragenyx Pharmaceutical Inc. ((RARE - Free Report) ) announced that the FDA has granted Orphan Drug Designation to UX701 for the treatment of Wilson disease. Wilson disease is a rare inherited disorder, in which excess copper builds up in the body. Its symptoms are typically related to the brain and liver.

Shares of the company have increased 201.7% year to date compared with the industry’s 5.9% growth.


Notably, orphan drug designation is granted to drugs that are capable of treating rare diseases that affect less than 200,000 people in the United States. This tag also makes the company entitled to certain other benefits including tax credits related to clinical trial expenses and an exemption from the FDA user fee.

UX701 is an investigational AAV type 9 gene therapy designed to deliver stable expression of the ATP7B copper transporter following a single intravenous infusion. It has been shown in preclinical studies to normalize copper trafficking and excretion from the body. The candidate is currently in late preclinical development and an investigational new drug application is expected to be filed with the FDA by 2020-end.

The available treatment involves complicated use of medications that block the absorption of copper from the diet or remove copper by chelation. However, UX701 is designed to directly address the underlying cause of the disease by restoring normal copper metabolism in the liver. Thus, this one-time treatment has a potential of better addressing the many serious effects of this disease.

The company also has some other 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).

Zacks Rank & Stocks to Consider

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

Some better-ranked stocks in the healthcare sector include ASLAN Pharmaceuticals Ltd. (ASLN - Free Report) , Aerpio Pharmaceuticals Ltd. (ARPO - Free Report) and Alimera Sciences Inc. (ALIM - Free Report) , each carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

ASLAN’s loss per share estimates have narrowed from 42 cents to 40 cents for 2020 and from 60 cents to 57 cents for 2021 over the past 60 days.

Aerpio’s loss per share estimates have narrowed from 15 cents to 14 cents for 2020 and from 63 cents to 57 cents for 2021 over the past 60 days.

Alimera’s loss per share estimates have narrowed from $1.31 to 96 cents for 2020 over the past 60 days.

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