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Horizon Pharma Gets FDA Nod for Procysbi Label Expansion

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Horizon Pharma plc (HZNP - Free Report) announced that the FDA has approved the label expansion for Procysbi (cysteamine bitartrate) delayed-release capsules. The label will include children aged one year and above suffering from nephropathic cystinosis. 

Procysbi is a delayed-release form of cysteamine bitartrate that works by continuously reducing the toxic concentration of cystine in the cells. It plays an important role in managing nephropathic cystinosis and is already approved for both the adults and minors as young as two year-olds. 

Horizon’s shares have underperformed the industry year to date. The stock has declined 7.2% against the industry’s growth of 3.5%.

The company’s receipt of the approval for Procysbi’slabel expansion was based on a study that enrolled 17 patients living with nephropathic cystinosis including 15 kids aged between one and five years, previously not treated with cysteamine therapy. The children experienced low white blood cell (WBC) cystine levels varying from poorly to well controlled stages at 12 and 18-month measurements. WBC levels are the biomarker for disease control.  . Also a marked improvement was noticed in their weight and height. The study was performed per the FDA’s requirement as a post-marketing commitment after Procysbi was approved.

The Procysbi prescribing information now contains an amended guidance for physicians administering the medicine to pediatric patients.

We remind investors that the ex-U.S. right to Procysbi was divested by Horizon. In May, the company had signed an agreement with Chiesi Farmaceutici S.p.A. to sell its European subsidiary — owning the marketing rights to Procysbi delayed-release capsules — and Quinsair (levofloxacin inhalation solution) in Europe, the Middle East and Africa (EMEA) regions.

Notably, both Procysbi and Quinsair were added in Horizon’s portfolio courtesy of the acquisition of Raptor Pharmaceutical Corp. in October 2016. This strategic transaction happened to strengthen Horizon’s orphan business in the United States and expand its footprint in Europe as well as in other key international markets.

However, following an evaluation of the commercial infrastructure, needed to make Procysbi and Quinsair available across the EMEA belt, the company decided to divest the rights in Europe.


Zacks Rank & Stocks to Consider

Horizon carries a Zacks Rank #3 (Hold). Some better-ranked health care stocks in the same space are Sucampo Pharmaceuticals , Achillion Pharmaceuticals, Inc. (ACHN - Free Report) and Corcept Therapeutics Incorporated (CORT - Free Report) , all carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Sucampo’s earnings per share estimates have moved up from 31 cents to $1.12 for 2017 and from $1.15 to $1.19 for 2018 in the last 30 days. The company delivered a positive earnings surprise in three of the trailing four quarters with an average beat of 15.63%. Share price of the company has surged 32.6% year to date.

Achillionhas seen the Zacks Consensus Estimate of current-year loss per share being narrowed from 65 cents to 63 cents and from 74 cents to 67 cents for 2018 in the last 60 days. The company came up with positive earnings surprises in two of the last four quarters with an average beat of 4.51%.

Corcept’s earnings per share estimates have been revised upward from 78 cents to 88 cents for 2018 in the last 60 days. The company pulled off positive earnings surprises in two of the trailing four quarters with an average beat of 14.32%. Shares of the company have skyrocketed 142.7% year to date.

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