MeiraGTx receives Orphan Drug Designation for A004 Gene Therapy for Retinitis Pigmentosa

Release Date: 24-Jan-2017

The US Food and Drug Administration (FDA) has granted orphan drug status to MeiraGTx’ s novel therapy treatment, A004 Gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP). A004 is the proprietary AAV-mediated gene therapy product candidate containing the retinitis pigmentosa GTPase regulator (RPGR) gene. The therapy works by targeting the RPGR gene to photoreceptor cells to restore function in RPGR-XLRP patients.


Retinitis pigmentosa (RP) is one of the most common forms of inherited blindness, with three general modes of inheritance: autosomal dominant, autosomal recessive and X-linked.  Over 70% of XLRP and up to 20% of all RP cases are caused by mutations in the RPGR gene.  Males with XLRP caused by mutations in RPGR (RPGR-XLRP) typically have night blindness in their first decade of life followed by progressive reduction of their visual field and loss of visual acuity.  By the end of their fourth decade, most patients are legally blind.
Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.  The OOPD provides incentives for sponsors to develop products for rare diseases, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. 


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