U.S. Markets closed
  • S&P 500

    4,395.64
    +41.45 (+0.95%)
     
  • Dow 30

    34,258.32
    +338.48 (+1.00%)
     
  • Nasdaq

    14,896.85
    +150.45 (+1.02%)
     
  • Russell 2000

    2,218.56
    +32.38 (+1.48%)
     
  • Gold

    1,768.40
    -9.80 (-0.55%)
     
  • Silver

    23.03
    +0.46 (+2.05%)
     
  • EUR/USD

    1.1696
    -0.0034 (-0.2924%)
     
  • 10-Yr Bond

    1.3360
    +0.0120 (+0.91%)
     
  • Vix

    20.87
    -3.49 (-14.33%)
     
  • GBP/USD

    1.3619
    -0.0045 (-0.3282%)
     
  • USD/JPY

    109.7800
    +0.5600 (+0.5127%)
     
  • BTC-USD

    41,746.42
    -1,085.53 (-2.53%)
     
  • CMC Crypto 200

    1,089.55
    +49.07 (+4.72%)
     
  • FTSE 100

    7,083.37
    +102.39 (+1.47%)
     
  • Nikkei 225

    29,639.40
    -200.31 (-0.67%)
     

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

  • Oops!
    Something went wrong.
    Please try again later.
  • Oops!
    Something went wrong.
    Please try again later.
  • Oops!
    Something went wrong.
    Please try again later.
·3 min read
In this article:
  • Oops!
    Something went wrong.
    Please try again later.
  • Oops!
    Something went wrong.
    Please try again later.
  • Oops!
    Something went wrong.
    Please try again later.

Ultragenyx Pharmaceutical Inc. (RARE) 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, Aerpio Pharmaceuticals Ltd. ARPO and Alimera Sciences Inc. ALIM, 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.

Ultragenyx Pharmaceutical Inc. Price

Ultragenyx Pharmaceutical Inc. Price
Ultragenyx Pharmaceutical Inc. Price

Ultragenyx Pharmaceutical Inc. price | Ultragenyx Pharmaceutical Inc. Quote

Just Released: Zacks’ 7 Best Stocks for Today

Experts extracted 7 stocks from the list of 220 Zacks Rank #1 Strong Buys that has beaten the market more than 2X over with a stunning average gain of +24.4% per year. These 7 were selected because of their superior potential for immediate breakout.

See these time-sensitive tickers now >>


Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report
 
Alimera Sciences, Inc. (ALIM) : Free Stock Analysis Report
 
Ultragenyx Pharmaceutical Inc. (RARE) : Free Stock Analysis Report
 
Aerpio Pharmaceuticals, Inc. (ARPO) : Free Stock Analysis Report
 
ASLAN Pharmaceuticals Ltd. (ASLN) : Free Stock Analysis Report
 
To read this article on Zacks.com click here.