First blepharoptosis drug gains FDA approval

The FDA has cleared a novel ophthalmic solution of oxymetazoline hydrochloride (Upneeq) for acquired blepharoptosis, making it the only approved therapy for the condition.

According to Osmotica Pharmaceuticals, once-daily application of the drug—a direct-acting alpha adrenergic receptor agonist—elevates the upper eyelid by selectively targeting the Müller’s muscle.

A pair of phase 3 trials showed that patients randomized to a daily dose of oxymetazoline 0.1% for 6 weeks experienced significant improvements in visual field and eyelid lift on day 1 and 14 compared with placebo. A third safety study demonstrated the treatment was well tolerated over a 12-week period, with mild and self-limiting adverse events. These events, which affected less than 5% of patients, included punctate keratitis, conjunctival hyperemia, dry eye, blurred vision, instillation site pain, eye irritation and headache.

“Upneeq’s safety and efficacy profile and its once-a-day dosing provides a significant ophthalmic therapeutic innovation,” said Tina DeVries, PhD, Osmotica’s executive vice president of research and development. “Given the previous absence of any approved medical treatment options, ptosis has been often under-diagnosed or overlooked. Upneeq has the potential to address a significant unmet need in ptosis therapy.”

Having already caught the interest of ophthalmic physicians and key opinion leaders, Osmotica plans to make the drug available to a selected group of eye care professionals via an early experience program. In addition, the company is engaging in active discussions to commercialize it beyond the United States.