Rare Daily Staff

The U. S. Food and Drug Administration granted rare pediatric disease designation to MeiraGTx’s gene therapy product candidate A002 for the treatment of patients with a rare inherited eye disorder.

A002 is an adeno-associated virus experimental gene therapy designed delivered to the back of the eye for expression in cone photoreceptors to treat achromatopsia due to mutations in the CNGB3 gene. Achromatopsia is a severe inherited retinal disorder characterized by severe vision loss, extreme light sensitivity, uncontrolled eye movements, and absence of color discrimination from birth. Currently, there are no effective treatments for this disease.

Achromatopsia is an inherited eye disorder that severely limits a person’s sight, as well as causing extreme light sensitivity, involuntary eye movement, and complete color blindness. A002 previously received orphan drug designation from the FDA and the European Medicines Agency for the treatment of achromatopsia in 2016.

FDA grants Rare Pediatric Disease Priority Review Vouchers to encourage the development of treatments for rare pediatric diseases. The vouchers are potentially lucrative because they are transferable and can be used to accelerate the review of large market therapeutics. The priority review voucher entitles the holder to priority review of a single New Drug Application or Biologics License Application.  Most recently Ultragenyx sold a rare pediatric priority review voucher it received for its MPSVII enzyme replacement therapy for $130 million.

MeiraGTx has dosed eight patients in an open label, multi-center phase 1/2 dose escalation trial of A002 in individuals diagnosed with achromatopsia due to biallelic mutations in CNGB3. The trial is currently treating patients in the highest of three dose cohorts. The primary endpoint of the study is to determine the safety of the treatment. Secondary endpoints include improvement in visual function including assessment of photophobia/photoaversion, retinal function, retinal structure and quality of life measures.

“With the receipt of our second rare pediatric disease designation in just two months, we have significant momentum for our ocular gene therapy pipeline as we begin 2018,” said Alexandria Forbes, president and CEO of MeiraGTx. “Achromatopsia is a severe and debilitating genetic disorder of the retina for which there are no effective treatment options.”

February 1, 2018
Photo: Alexandria Forbes, CEO of MeiraGTx

X