NEW YORK – MeiraGTx on Friday said it intends to submit a marketing authorization application (MAA) to the UK Medicines and Healthcare products Regulatory Authority (MHRA) for its investigational gene therapy for Leber congenital amaurosis 4 (LCA4), known as rAAV8.hRKp.AIPL1, based on data from 11 treated children.
MeiraGTx will submit the MAA under exceptional circumstances, a type of marketing authorization granted in cases where a sponsor is unable to provide comprehensive efficacy and safety data, such as in rare diseases. The New York-based biotech said it aligned with the MHRA on an expedited chemistry, manufacturing, and controls package to support such an approval.
Separately, MeiraGTx is also engaged with the US Food and Drug Administration to discuss a potential pathway for expedited approval in the US.
"We are incredibly excited to see the transformative effect of treatment with rAAV8.hRKp.AIPL1 in every one of the young children who received this genetic medicine," MeiraGTx President and CEO Alexandria Forbes said in a statement. "We are working with global regulators on expedited approval of rAAV8.hRKp.AIPL1."
The gene therapy aims to restore vision in patients with LCA4, a severe form of inherited blindness caused by mutations in the AIPL1 gene. The treatment is administered through a one-time subretinal injection that delivers functional copies of AIPL1 to cone and rod photoreceptors in the central retina.
Investigators reported results from the first four children treated in the company's open-label, first-in-human study in the UK in a paper published in the Lancet this weekend. For the study, patients between the ages of 1 and 3 years old received an injection in one eye and since then have experienced improved visual acuity and functional vision in those treated eyes.
Seven additional patients received the gene therapy in both eyes with similar outcomes, according to MeiraGTx. In total, all 11 of the patients, all of whom were blind at birth, have gained improvements in vision after at least four weeks posttreatment.
In the EU, MeiraGTx's gene therapy has been granted orphan designation by the European Commission and also has received orphan drug and rare pediatric disease designations from the FDA in the US.