FDA sets goal date for ophthalmic version of bevacizumab

The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of July 29, 2026, for Outlook Therapeutics’ resubmitted application of ONS-5010/Lytenava (bevacizumab-vikg). If approved, it would be used to treat patients with neovascular age-related macular degeneration (nAMD), or wet AMD.

Age-related macular degeneration occurs when the macula, part of the retina at the back of the eye, becomes damaged. About 10% of all cases become wet, which leads to vision loss as a result of excess blood vessel growth between two layers of cells in the retina.

Lytenava is an ophthalmic formulation of bevacizumab, a recombinant humanized monoclonal antibody, and it inhibits vascular endothelial growth factor (VEGF), blocking blood vessel growth. It is administered through an intravitreal injection, which is placed in the space in the back of the eye. If approved, Lytenava would be the only bevacizumab product to specifically treat AMD.

Bevacizumab is available as Avastin — along with six U.S.-approved biosimilars — to treat patients with cancers such as colorectal, lung, glioblastoma, kidney, cervical, and ovarian cancer. It is used off-label to treat wet AMD; when used for the eye, bevacizumab has to be obtained from compounding pharmacies, which repackage small quantities into syringes for intravitreal injection.

About 2.7 million injections of off-label bevacizumab are given in the United States, according to an investor presentation from Outlook Therapeutics officials in May 2026. About 55% of these injections are used as first-line treatment, and 34% are used as maintenance therapy.

“ONS-5010/Lytenava has the potential to offer clinicians and patients an important additional option for treating wet AMD. If approved as the first on-label ophthalmic formulation of bevacizumab, ONS-5010 will enable patients to benefit from wider access and provide an attractive alternative for anti-VEGF treatment of wet AMD,” Baruch Kuppermann, M.D., Ph.D., Steinert Endowed Professor, Chair, department of Ophthalmology and Visual Sciences, and director, Gavin Herbert Eye Institute University of California, Irvine, said in a news release when Outlook Therapeutics had announced the resubmission of the BLA.

In December 2025, the FDA issued its third complete response letter for Lytenava. Regulatory officials recommended that confirmatory evidence of efficacy be submitted. They noted that the additional mechanistic and natural history data information provided in the resubmitted biological license applications (BLA) does not change the agency's previous review conclusion.

Outlook Therapeutics officials said the company had successfully appealed the December 2025 complete response letter related to the BLA. In their decision, the FDA Office of New Drugs concluded that substantial evidence of effectiveness has been established for the treatment of neovascular age-related macular degeneration and additional trials are not required.

Officials with the Office of New Drugs has directed the FDA’s Division of Ophthalmology and Office of Specialty Medicine to work with Outlook Therapeutics to reach an agreement on final labeling.

The U.S. BLA is based on data from the complete data set from the NORSE clinical trial program, which included the NORSE TWO pivotal clinical trial, as well as safety and efficacy data from all other NORSE trials. The resubmission also included data from NORSE EIGHT, a non-inferiority study evaluating Lytenava compared with Lucentis (ranibizumab) in a 12-week study of treatment-naive patients with a primary efficacy endpoint at 8 weeks.