Ocuphire Pharma, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for Nyxol (phentolamine ophthalmic solution 0.75%) for the treatment of pharmacologically-induced mydriasis (RM). The FDA assigned a Prescription Drug User Fee Act (PDUFA) date of September 28, 2023.
“The FDA’s acceptance of the NDA submission and PDUFA date in late September for Nyxol sets the stage for an exciting 2023”, said Mina Sooch, MBA, founder and CEO of Ocuphire Pharma. “We look forward to working closely with the FDA during the review process. If approved later this year, Nyxol eye drops will be the first prescription reversal drop available for patients in the US. In addition, we look forward to leveraging the synergies of this first NDA for Nyxol in potential supplementary NDAs for presbyopia and dim light vision disturbances indications in the future.”
Paul Karpecki, OD, FAAO, clinical director of Cornea at Kentucky Eye Institute, stated “Over 100 million eye dilations are performed each year in the US in pediatric and adult patients, and we expect dilations, which are the standard of care for comprehensive eye exams, to increase due to an aging population. Patients often express frustration and discomfort with the effects of prolonged dilation and eye care professionals underestimate the time it takes for patients to return to normal pupil size. A reversal drop would significantly improve patient experience and productivity.”
The NDA filing is supported by positive results from the comprehensive MIRA clinical program collectively involving over 600 subjects, including the MIRA-1 phase 2b trial, MIRA-2 and MIRA-3 phase 3 pivotal trials, and MIRA-4 phase 3 pediatric trial.
The MIRA-2 and MIRA-3 trials successfully met their primary and key secondary endpoints, demonstrating statistically significant superiority of Nyxol compared to placebo to rapidly return dilated eyes to their baseline pupil diameter as early as 60 and 90 minutes. Nyxol consistently showed a favorable safety and tolerability profile across all trials. In addition, the positive MIRA-4 pediatric trial results support a potential broader label for Nyxol in RM to include subjects aged 3 and older.
About Nyxol
Nyxol is a proprietary, preservative-free, stable, investigational eye drop formulation of phentolamine ophthalmic solution 0.75% designed to uniquely modulate the pupil size by blocking the α1 receptors found only on the iris dilator muscle without affecting the ciliary muscle. Nyxol is being developed for reversal of pharmacologically-induced mydriasis (RM), presbyopia, and dim light (night) vision disturbances (DLD) under the 505(b)(2) pathway.
Nyxol has been studied in a total of 12 clinical trials (3 phase 1, 5 phase 2, 4 phase 3) in a total of approximately 1,100 patients (with over 650 Nyxol-treated) and has demonstrated promising clinical data for use in the multiple ophthalmic indications mentioned above. Ocuphire reported positive efficacy data from the MIRA-1, MIRA-2, MIRA-3 and MIRA-4 trials in RM, Phase 3 LYNX-1 trial in DLD and Phase 2b VEGA-1 trial for Nyxol as single agent and as adjunctive therapy with 0.4% low dose pilocarpine in presbyopia.
About Reversal of mydriasis
An estimated 100 million eye dilations are conducted every year in the U.S. to examine the back of the eye either for routine check-ups, disease monitoring or surgical procedures across all eye care practice groups. Depending on the individual and the color of their eyes, the pharmacologically-induced dilation can last anywhere from 6 to 24 hours in adults and children.
Dilated eyes have heightened sensitivity to light and an inability to focus on near objects, causing difficulty reading, working, and driving. Currently, there are no approved treatment options to reverse mydriasis.
If approved, Nyxol has the potential to be the first and only FDA-approved agent for the reversal of mydriasis uniquely modulating the iris dilator muscle.
Market research conducted by GlobalData surveyed several hundred patients and eye care providers (optometrists and ophthalmologists) about reversal of mydriasis. Over 65% of surveyed patients reported moderate to severe negative impact of a dilated pupil. These data underscore the potential value of the role of the product candidate Nyxol in improving comfort and daily function after pupil dilation.
Furthermore, approximately 80% of patients responded that they would be likely to request a dilation reversal drop, and more than 70% of eye care providers would be likely to use a reversal drop. The market research confirmed patients’ willingness to pay out-of-pocket to reverse their dilations.