MeiraGTx Announces AAV-CNGA3 Has Received Orphan Drug Designation

MeiraGTx Holdings Plc recently announced that the US FDA has granted orphan drug designation (ODD) for its AAV-CNGA3 gene therapy product candidate for the treatment of achromatopsia (ACHM) caused by mutations in the CNGA3 gene.

ACHM is an inherited retinal disease that severely limits a person’s sight by preventing cone photoreceptors in the eye from functioning. Individuals with ACHM are often legally blind from birth, have extreme sensitivity to light, and experience involuntary eye movements. AAV-CNGA3 is an investigational gene therapy treatment designed to restore cone function, delivered to the cone receptors at the back of the eye via subretinal injection.

“Without any currently approved therapies, we are very pleased by the FDA’s decision and the recognition from the Agency that those suffering from ACHM are in need of urgent treatment options,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “This designation is the second important regulatory milestone we’ve received for AAV-CNGA3 in just two months and we look forward to continuing the momentum in this program for those in need of relief from this debilitating disease.”

In June 2018, the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products issued a positive opinion recommending orphan medicinal product (orphan drug) designation for AAV-CNGA3 for the treatment of ACHM. In addition to AAV-CNGA3, MeiraGTx also has orphan designation in both the U.S. and EU for three other inherited retinal disease gene therapy product candidates, AAV-CNGB3, AAV-RPGR and AAV-RPE65, all of which are in clinical development.

Orphan drug designation is intended to facilitate and expedite drug development for rare diseases or conditions for which there are no current treatments available. It also provides substantial benefits to the sponsor, including the potential for tax credits for clinical development costs and study-design assistance. If a product receives the first FDA approval for the indication for which it has orphan drug designation, the product is entitled to seven years of market exclusivity, except in limited circumstances.

Achromatopsia is an inherited retinal disorder that specifically prevents cone photoreceptors from functioning. ACHM is characterized by severely reduced visual acuity of 20/200 or worse, disabling light sensitivity (photoaversion) and involuntary back and forth eye movements (nystagmus). ACHM occurs in approximately one in 30,000 people in the United States, with 92 percent of cases caused by mutations in CNGB3 and CNGA3 genes. Currently, there are no effective treatments for this disease.

MeiraGTx (NASDAQ:MGTX) is a vertically integrated, clinical stage gene therapy company with four ongoing clinical programs and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, severe forms of xerostomia and neurodegenerative diseases. Though initially focusing on the eye, salivary gland and central nervous system, MeiraGTx intends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases. For more information, visit www.meiragtx.com.