AGTC announces U.S. FDA Orphan Drug designation for gene therapy to treat X-linked retinitis pigments.
Applied Genetic Technologies Corporation, a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, announced that the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation for its gene therapy product candidate for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene. In June 2016 the company announced that the European Commission (EC) granted orphan medicinal product designation for the same indication.
XLRP is an inherited condition that causes progressive vision loss, beginning with night blindness in young boys followed by progressive constriction of the field of vision. Affected men become legally blind at an average of about 45 years of age. The most common form of XLRP is caused by mutations in the RPGR gene.
Preclinical data indicate that treatment with a gene therapy product slowed the loss of visual function in a canine model of XLRP due to mutations in the RPGR gene. XLRP and X-linked retinoschisis (XLRS) are the two development programs within AGTC’s collaboration and license agreement with Biogen Inc., to develop gene-based therapies for multiple ophthalmic diseases.
“Receiving U.S. orphan drug designation for our fourth gene therapy treatment candidate demonstrates our ongoing commitment to addressing the unmet needs of people with rare inherited diseases,” said Sue Washer, president and CEO of AGTC. “XLRP affects approximately 20,000 people in the U.S. and Europe. We are on track to file an Investigational New Drug application for this program this year, and this orphan drug designation will help to accelerate the regulatory review process as we work to bring a new treatment option to XLRP patients.”
AGTC has also been granted orphan drug designation from the FDA and EC for its gene therapy product candidates for the treatment of X-linked retinoschisis and for the treatment of achromatopsia caused by mutations in the CNGA3 and CNGB3 genes.
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.
(Source: GlobeNewswire)
