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Horizon's NDA for Procysbi New Dosage Form Accepted by FDA

Zacks Equity Research

Horizon Therapeutics plc (HZNP announced that the FDA has accepted its new drug application (NDA) for Procysbi (Cysteamine Bitartrate) delayed-release oral granules in packets. Procysbi is a delayed-release capsule 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 adults and minors as young as one-year olds. 

If the FDA grants approval, Procysbi’s new dosage form would be another option for patients, in addition to the currently available Procysbi delayed-release capsules.

The FDA is expected to make a decision on the approval of the proposed new dosage form in 2020. Nephropathic cystinosis is a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues and organs in the body. 

The proposed dosage will provide patients with the option of opening a packet containing small, compact granules, and sprinkling those on certain foods and juice. These packets would be an alternative to the currently available option of opening individual capsules to remove the granules.

Shares of the company have rallied 26% year to date compared with the industry’s growth of 1.7%.

We note thatProcysbi was added in Horizon’s portfolio, courtesy of the acquisition of Raptor Pharmaceutical Corp. in October 2016. This transaction was made to strengthen Horizon’s orphan business in the United States, and expand its footprint in Europe and other key international markets.

However, in May 2017, the company signed an agreement with Chiesi Farmaceutici S.p.A. to sell its European subsidiary that owned the marketing rights to Procysbi delayed-release capsules in Europe, the Middle East and Africa (EMEA) regions. 

 

Horizon Pharma Public Limited Company Price

 

Horizon Pharma Public Limited Company Price

Horizon Pharma Public Limited Company price | Horizon Pharma Public Limited Company Quote

Zacks Rank & Stocks to Consider

Horizon Pharma currently has a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Alnylam Pharmaceuticals Inc. ALNY, Applied Genetics Technologies Corp. AGTC and Acorda Therapeutics Inc. ACOR. While Alnylam sports a Zacks Rank #1 (Strong Buy), Applied Genetics and Acorda carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Alnylam’s loss per share estimates have narrowed from $6.90 to $6.43 for 2020 in the past 60 days. The company delivered a positive earnings surprise in three of the trailing four quarters, with average beat of 8.93%.    

Applied Genetics’ loss per share estimates have narrowed from $2.39 to $2.15 for 2020 in the past 90 days. The company delivered a positive earnings surprise in three of the trailing four quarters, with average beat of 83.47%.    

Acorda’s loss per share estimates have narrowed from $3.16 to $3.05 for 2020 in the past 60 days. The company delivered a positive earnings surprise in the trailing four quarters, with average beat of 79.32%.    

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