SEP 24, 2018
The FDA awarded fast track designation to a single-administration intravitreal gene therapy for wet AMD, according to a press release by Adverum Biotechnologies.
The treatment, ADVM-022, uses a proprietary adeno-associated virus vector that carries an aflibercept coding sequence. The company hopes that the therapy will provide sustained VEGF inhibition to minimize the injection burden in patients with wet AMD.
The open-label phase 1 trial that will test the safety and tolerability of the drug is scheduled to begin later this year. Investigators plan to enroll 18 wet AMD patients who are responsive to anti-VEGF treatment at multiple U.S. centers, where they will receive 3 escalating doses with a tapering prophylactic corticosteroid regimen. The primary safety endpoint will be assessed at 24 weeks, along with BCVA, central retinal thickness and the number of rescue aflibercept injections. The study will follow patients for a total of 2 years.
Earlier this year, findings from a primate study hinted that ADVM-022’s long-term efficacy was comparable to aflibercept. Thirteen months after injection, the therapy appeared safe and significantly prevented grade 4 lesions compared with a vehicle control. Furthermore, researchers noted “robust” levels of aflibercept in the aqueous and vitreous humor and in the retina and choroid tissues for up to 16 months after injection.
“The FDA’s fast track designation is an important recognition of our ADVM-022 gene therapy program,” stated Leone Patterson, CEO of Adverum Biotechnologies. “We look forward to working with the FDA and benefiting from the potential expedited development and regulatory path offered by their fast track program.”