Horizon's NDA For Procysbi New Dosage Form Accepted By FDA
Zacks Investment Research | Jul 18, 2019 09:41PM ET
Horizon Therapeutics plc ( (NASDAQ: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 Zacks Investment Research
Get The News You Want
Trading in financial instruments and/or cryptocurrencies involves high risks including the risk of losing some, or all, of your investment amount, and may not be suitable for all investors. Prices of cryptocurrencies are extremely volatile and may be affected by external factors such as financial, regulatory or political events. Trading on margin increases the financial risks.
Before deciding to trade in financial instrument or cryptocurrencies you should be fully informed of the risks and costs associated with trading the financial markets, carefully consider your investment objectives, level of experience, and risk appetite, and seek professional advice where needed.
Fusion Media would like to remind you that the data contained in this website is not necessarily real-time nor accurate. The data and prices on the website are not necessarily provided by any market or exchange, but may be provided by market makers, and so prices may not be accurate and may differ from the actual price at any given market, meaning prices are indicative and not appropriate for trading purposes. Fusion Media and any provider of the data contained in this website will not accept liability for any loss or damage as a result of your trading, or your reliance on the information contained within this website.
It is prohibited to use, store, reproduce, display, modify, transmit or distribute the data contained in this website without the explicit prior written permission of Fusion Media and/or the data provider. All intellectual property rights are reserved by the providers and/or the exchange providing the data contained in this website.
Fusion Media may be compensated by the advertisers that appear on the website, based on your interaction with the advertisements or advertisers.