MeiraGTx Receives Positive Opinion for Orphan Drug Designation in the European Union for AAV-CNGA3 for the Treatment of Achromatopsia
June 26, 2018
LONDON and NEW YORK, June 26, 2018 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq:MGTX), a vertically integrated, clinical stage gene therapy company, today announced that the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products issued a positive opinion recommending orphan medicinal product (orphan drug) designation of AAV-CNGA3 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.
“We are encouraged by this positive opinion from the EMA and believe it’s an important step in bringing a much-needed potential therapy to patients with ACHM, who suffer from debilitating symptoms and face limited therapeutics options,” said Stuart Naylor, Ph.D., Chief Development Officer of MeiraGTx. “We look forward to working closely with the EMA and continuing the clinical development of AAV-CNGA3 for those in need.”
Orphan drug designation by the European Commission provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to centralized marketing authorization.
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, please visit www.meiragtx.com
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