Ocuphire Pharma receives PDUFA fee waiver for NDA from FDA

Ocuphire Pharma Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted a small business waiver of the Prescription Drug User Fee Act (PDUFA) fee of $3.1 million for the 505(b)(2) New Drug Application (NDA) for phentolamine ophthalmic solution (Nyxol), the company’s late stage product candidate. Ocuphire remains on track to file the NDA for phentolamine ophthalmic solution in its first indication, reversal of mydriasis (RM), in late 2022.

“We are pleased to receive this meaningful NDA fee waiver for Nyxol and look forward to continuing to work with the FDA throughout the submission and review process,” said Mina Sooch, MBA, founder and CEO of Ocuphire Pharma. “NDA submission in late 2022 is a high priority. In parallel, we are ramping up our pre-commercial activities and continue discussions with commercial partners in preparation for the anticipated approval and launch of Nyxol in 2023 as potentially the only dilation reversal drops.”

According to the company, 0.75% phentolamine ophthalmic solution is a once-daily, preservative-free eye drop formulation of phentolamine mesylate, a non-selective alpha-1 and alpha-2 adrenergic antagonist designed to reduce pupil size, and is being developed for several indications, including reversal of pharmacologically-induced mydriasis (RM), presbyopia and dim light or night vision disturbances (NVD).

Phentolamine ophthalmic solution has been studied in 12 completed clinical trials, with positive data reported from the MIRA-2 and MIRA-3 registration trials and the MIRA-4 pediatric safety trial for the treatment of RM. Ocuphire also reported positive top-line data from the VEGA-1 Phase 2 trial of phentolamine ophthalmic solution for treatment of presbyopia, which evaluated both phentolamine ophthalmic solution as a single agent and phentolamine ophthalmic solution with low dose pilocarpine (LDP) 0.4% as adjunctive therapy.

The company announced positive top-line results from the LYNX-1 Phase 3 trial of phentolamine ophthalmic solution for night vision disturbances (NVD).

Ocuphire’s second product candidate, APX3330, is an oral tablet designed to inhibit angiogenesis and inflammation pathways relevant to retinal and choroidal vascular diseases, such as diabetic retinopathy (DR) and diabetic macular edema (DME). APX3330t has been studied in 11 Phase 1 and 2 trials. The company announced the completion of last patient last visit in late August with top-line results expected in 4Q22.

Ocuphire has an ongoing APX3330 Phase 2b trial in DR/DME ZETA-1 (NCT04692688) and completed Nyxol trials, including Phase 3 registration trial in NVD LYNX-1 (NCT04638660), Phase 3 registration trials in RM MIRA-2 (NCT04620213) and MIRA-3 (NCT05134974), MIRA-4 Phase 3 pediatric safety study (NCT05223478), and Phase 2 trial in presbyopia VEGA-1 (NCT04675151).