Axovant's GM1 Gangliosidosis Candidate Gets Orphan Drug Tag
Zacks Investment Research | Nov 18, 2019 09:40PM ET
Axovant Gene Therapies Ltd. (NASDAQ:AXGT) announced that the FDA has granted an Orphan Drug designation to its investigational gene therapy candidate, AXO-AAV-GM1, being developed in a phase I/II study for the potential treatment of GM1 gangliosidosis, a rare, inherited neurodegenerative lysosomal storage disorder.
Notably, the Orphan Drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States. This status also makes the company entitled to certain other benefits, including tax credits, clinical study expenses and a seven-year market exclusivity upon potential approval from the regulatory body.
Despite the positive news, shares of Axovant declined 4.9% on Monday. In fact, the stock has plunged 43.9% so far this year against the Original post
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.