Adverum’s lead gene therapy candidate, ADVM-022, is a one-time, intravitreal (IVT) injection for the treatment of patients with wet AMD.
PRIME is a program launched by the EMA to enhance support for research on and development of medicines that target a significant unmet medical need. This regulatory program offers developers of promising medicines enhanced interaction and early dialogue and is designed to optimize development plans and speed evaluation ensuring these medicines reach patients as early as possible.
According to the EMA, developers of medicines that are eligible for PRIME can expect additional opportunities for scientific advice and be eligible for accelerated assessment at the time of application for a marketing authorization. Currently, only three ophthalmology therapies to date have been granted PRIME designation out of 14 submissions.
"We are extremely pleased with EMA's decision to grant ADVM-022 PRIME designation. Following the encouraging results seen in the OPTIC trial in wet AMD, our PRIME designation recognizes the broad potential of ADVM-022 and is based in part on our trial subjects now having 3-year sustained aflibercept expression with stable disease and maintained long-term central subfield thickness and visual acuity after a single IVT injection," Laurent Fischer, MD, president and chief executive officer at Adverum Biotechnologies, said in a company news release.
“The current standard of care in wet AMD, a highly prevalent disease, requires frequent anti-vascular endothelial growth factor (anti-VEGF) injections in the eye and is a lifelong burden for many patients and their caregivers. We believe ADVM-022 has the potential to provide a durable, safe and cost-effective in-office treatment option that addresses the needs of these patients, and their families, as well as retina specialists and health systems worldwide. We look forward to taking advantage of the benefits provided to us through PRIME as we continue the development of ADVM-022 in areas of high unmet need,” Mr. Fischer added.
A phase 2 study in wet AMD is planned to investigate the 2x10^11 vg/eye dose and a new, lower 6x10^10 vg/eye dose of ADVM-022, as well as new enhanced prophylactic steroid regimens, including local steroids and a combination of local and systemic steroids. Adverum anticipates dosing the first patient in the third quarter of 2022.