Sign up USA
Proactive Investors - Run By Investors For Investors

AGTC granted FDA orphan drug designation for gene therapy to treat achromatopsia

Achromatopsia is an inherited retinal disease caused by mutations in the CNGA3 gene.
AGTC granted FDA orphan drug designation for gene therapy to treat achromatopsia
Products receiving orphan drug designation are eligible to receive market exclusivity for a period of seven years

Applied Genetic Technologies (NASDAQ:AGTC), a biotechnology company focused on rare eye diseases, said the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation for its gene therapy product candidate for the treatment of achromatopsia, an inherited retinal disease, caused by mutations in the CNGA3 gene.

AGTC is developing products for achromatopsia based on mutations in the CNGA3 and CNGB3 genes, which together account for approximately 75% of the total achromatopsia patient population.

The Alachua, Florida-based company previously announced that the European Medicines Agency (EMA) granted orphan designation for the same indication in October.

"Receiving U.S. Orphan Drug designation is another significant milestone as we continue to advance our pipeline of novel gene therapies to treat rare inherited eye disorders," said Sue Washer, CEO of AGTC.

AGTC presented early preclinical findings in a naturally occurring dog model of the CNGB3 form of the disease, and previously received orphan drug designation from the FDA and the EMA for its investigational gene therapy product for the treatment of an additional variant of achromatopsia caused by mutations in the CNGB3 gene.

Orphan drug designation, covered by the U.S. Orphan Drug Act of 1983, is granted to drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 individuals.

Products receiving orphan drug designation are eligible to receive market exclusivity for a period of seven years, an accelerated regulatory review process, an exemption from certain taxes and user fees and additional clinical support from FDA.

 

View full AGTC profile View Profile

AGTC Timeline

Related Articles

People working in a laboratory
May 02 2018
The benefits of Admedus' proprietary ADAPT technology and products are now scientifically proven beyond doubt.
mri scanner
September 12 2018
IXICO is actively supporting the hunt for breakthrough treatments for Alzheimer’s, Parkinson’s, Huntington’s and multiple sclerosis
test tubes
September 11 2018
Sierra Oncology is about to start the third trial of Sareum’s SRA737 Chk1 inhibitor, while internally, Sareum is making “good progress” with its TYK2 inhibitor programme

No investment advice

The Company is a publisher. You understand and agree that no content published on the Site constitutes a recommendation that any particular security, portfolio of securities, transaction, or investment strategy is suitable or advisable for any specific person. You understand that the Company receives either monetary or securities compensation for our services. We stand to benefit from any volume this write-up may generate.

You further understand that none of the information providers or their affiliates will advise you personally concerning the nature, potential, advisability, value or suitability of any particular security, portfolio of securities, transaction, investment strategy, or other matter.

You understand that the Site may contain opinions from time to time with regard to securities mentioned in other products, including company related products, and that those opinions may be different from those obtained by using another product related to the Company. You understand and agree that contributors may write about securities in which they or their firms have a position, and that they may trade such securities for their own account. In cases where the position is held at the time of publication and such position is known to the Company, appropriate disclosure is made. However, you understand and agree that at the time of any transaction that you make, one or more contributors may have a position in the securities written about. You understand that price and other data is supplied by sources believed to be reliable, that the calculations herein are made using such data, and that neither such data nor such calculations are guaranteed by these sources, the Company, the information providers or any other person or entity, and may not be complete or accurate.

From time to time, reference may be made in our marketing materials to prior articles and opinions we have published. These references may be selective, may reference only a portion of an article or recommendation, and are likely not to be current. As markets change continuously, previously published information and data may not be current and should not be relied upon.

Copyright © Proactiveinvestors.com, 2018. All Rights Reserved - Proactive Investors North America Inc., Proactive Investors LLC

Market Indices, Commodities and Regulatory News Headlines copyright © Morningstar. Data delayed 15 minutes unless otherwise indicated. Terms of use