Last month, the US Food and Drug Administration approved a novel ophthalmic solution to treat acquired blepharoptosis, otherwise known as “droopy lid syndrome.” The solution, Osmotica Pharmaceuticals‘ Upneeq (oxymetazoline hydrochloride 0.1%, formerly known as RVL-1201), is a first-in-class agent and the only approved drug indicated for droopy upper eyelids, which can interfere with patients’ field of vision.
Now, Osmotica is taking a unique approach to building out its distribution by setting up a separate wholly owned subsidiary.
Normally, the Müller muscle along with the levator superioris acts to elevate the eyelid. In patients with ptosis, however, that muscle is partially or completely dysfunctional. Oxymetazoline is a direct-acting alpha adrenergic receptor agonist, which, when administered to the eye, is believed to selectively target the Müller muscle and elevate the upper eyelid. Until now, most patients struggling with droopy lids had surgery as their only corrective option.
Osmotica reports that “millions of people have ptosis in the United States alone, and an even larger population may be affected in Europe, Japan, and China.” One study of adults age 50 and older indicated a prevalence of about 11.5%. Osmotica estimates ptosis affects about 17 million individuals in the US; about 2.5 million of them have been diagnosed or have discussed the condition with a physician.
Upneeq offers a non-surgical option to treat ptosis, potentially addressing a significant unmet need. “Upneeq’s safety and efficacy profile and its once-a-day dosing provide a significant ophthalmic therapeutic innovation,” said Tina deVries, PhD, executive VP for research and development. “Given the previous absence of any approved medical treatment options, ptosis has been often underdiagnosed or overlooked.”
FDA approval was based on two six-week randomized, double-masked, placebo-controlled Phase III efficacy studies of 304 patients that found oxymetazoline was associated with significant improvements compared with placebo in primary superior visual field and eyelid lift.
The primary efficacy endpoint was change in the upper visual field from baseline to treatment day 1 (six hours after instillation), and treatment day 14 (two hours after instillation). The secondary efficacy endpoint, change from baseline as measured by the marginal reflex distance test, was assessed at screening and treatment days 1, 14, and 42 (end of the study).
A third randomized, multicenter, double-masked, placebo-controlled safety study (n=234) showed that Upneeq was well tolerated over 12 weeks when administered once daily in the morning. Osmotica noted that the majority of adverse events were mild and self-limited.
Getting Prescribers Interested
“Upneeq has garnered a great deal of interest from ophthalmic physicians and key opinion leaders … and we are prepared to commercialize Upneeq and engage providers through our medical education outreach,” CEO Brian Markison said.
The company will launch the product from its subsidiary, RVL Pharmaceuticals, which was created solely to bring Upneeq to market. It will be made available exclusively through Osmotica’s fully owned and integrated pharmacy. Markison added that the firm is looking for international partners with whom to commercialize the product outside the US.
The company seeks to provide unrestricted access to Upneeq for all patients and providers “through a fully transparent cash pricing model that removes insurance barriers and hassles from the prescribing process.”
Osmotica’s product-approval webcast stated the therapy will cost $3 to $4 per dose for 30 doses for a total prescription price of $90 to $120 per month. The program will offer “optimal value” with a 25% to 35% discount for a 90-day supply and “downstream flexibility to introduce a buy-and-bill pricing model.”
The initial focus of Osmotica’s launch will target approximately 650 leading eye-care practices, building to more than 2,500 practices, while tasking its sales team with establishing the product’s “first and only” position and robust safety and efficacy profile.
RVL Pharmaceuticals is more than a distribution arm for Osmotica. The subsidiary entered into an exclusive license agreement with Santen Pharmaceutical covering the development, registration and commercialization rights in Japan, China, and other Asian countries as well as Europe, Middle East, and Africa to RVL-1201. Santen will be responsible for further development of RVL-1201 and regulatory approvals as well as commercialization in its licensed territories under the agreement.