Outlook Therapeutics Inc (NASDAQ:OTLK) said the US Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for its investigational ophthalmic formulation of bevacizumab for treating wet age-related macular degeneration (wet AMD).
The FDA set a Prescription Drug User Fee Act (PDUFA) goal date of 29 August 2023. If approved, Outlook Therapeutics said its drug labelled ONS-5010 – brand name Lytenava – is expected to receive 12 years of regulatory exclusivity in the US.
Outlook Therapeutics CEO Russell Trenary said the BLA acceptance and PDUFA date are significant milestones in the Iselin, New Jersey-based company’s mission to offer clinicians and their patients the first and only on-label ophthalmic bevacizumab to treat wet AMD.
READ: Outlook Therapeutics enters into commercialization agreement with AmerisourceBergen
"If approved, ONS-5010 (bevacizumab-vikg) will meet the robust FDA requirements for use as an ophthalmic injectable. We estimate that approximately 50% of the millions of anti-VEGF injections in ophthalmology are off-label compounded bevacizumab that is administered with no FDA-approved labelling,” Trenary said in a statement.
Originally used as a cancer treatment, off-label bevacizumab does not meet the same standards Outlook Therapeutics said it must achieve to earn FDA approval in ophthalmology. Repackaged IV bevacizumab provided by compounding pharmacies has known risks of contamination and inconsistent potency and availability, the company said.
“We hope to enhance the standard of care in the anti-VEGF space, and we are proud of the entire team and our clinical partners and advisors for bringing ONS-5010 to this point in its journey towards FDA approval," said Trenary.
Outlook Therapeutics COO Terry Dagnon said he’s gratified the FDA has begun its review of the application.
"ONS-5010 is designed and manufactured to be fully compliant with the FDA's criteria for ophthalmic intravitreal biologics and we are excited about the prospect of filling the public health need for an FDA-approved ophthalmic formulation of bevacizumab," Dagnon said.
As part of the company's multi-year commercial planning process, and in anticipation of potential FDA approval in August 2023, Outlook Therapeutics and AmerisourceBergen recently announced a strategic commercialization agreement to expand the company's reach.
AmerisourceBergen will provide third-party logistics services and distribution, as well as pharmacovigilance services in the US. Through this agreement, Outlook Therapeutics said it expects to increase market access and efficient distribution of ONS-5010, while not duplicating services provided by AmerisourceBergen, thereby decreasing Outlook's expense for those services.
Outlook Therapeutics said it also established partnerships with FUJIFILM Diosynth Biotechnologies for drug substance manufacturing and with drug product manufacturer Ajinomoto Bio-Pharma Services for the finished drug product.
In addition to the US filing, Outlook Therapeutics said it is developing registration documents on a parallel path for approvals in Europe and expects to submit them in the fourth quarter of 2022. Outlook Therapeutics said it is assessing direct commercialization and partnerships in Europe and Asia on a country-by-country basis.
Norse clinical program for wet AMD
Outlook Therapeutics said it has conducted three clinical trials – Norse One, Two and Three – for use of ONS-5010 in patients in the US and Australia.
Norse One was a clinical experience trial involving 61 wet AMD participants in Australia. The trial compared ONS-5010 to ranibizumab (brand name Lucentis) as a treatment for wet AMD and showed the first markers of efficacy and safety in humans, according to the company.
Norse Two enrolled 228 wet AMD patients in the US and showed that 41.7% of patients gained 15 letters of vision on an eye chart test, 56.5% gained 10 letters of vision and 68.5% gained five letters. The company said the patients who received ranibizumab only gained an average of 5.8 letters on the eye chart.
Norse Three was an open-label safety study of ONS-5010 in 197 patients in the US, to provide the necessary number of retina patients dosed with ONS-5010 to complete the safety requirements for the BLA submission, the company said.
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