Industry leaders react to FDA approval of perfluorohexyloctane ophthalmic solution for treatment of dry eye disease

Perfluorohexyloctane ophthalmic solution is the first and only FDA-approved treatment for DED that directly targets tear evaporation. (Image courtesy of Adobe Stock)

The FDA this week announced its approval of the perfluorohexyloctane ophthalmic solution, formerly known as NOV03 (MIEBO, Bausch + Lomb Corp. and Novaliq GmbH) for the treatment of dry eye disease.

According to the companies, perfluorohexyloctane ophthalmic solution is the first and only FDA-approved treatment for DED that directly targets tear evaporation.¹

Brent Saunders, chairman and CEO of Bausch + Lomb, noted that the approval of perfluorohexyloctane ophthalmic solution advances DED treatment by addressing a significant unmet need for millions of Americans suffering with the symptoms of the disease.

“We are proud to bring to market the first and only prescription eye drop approved in the United States for the treatment of DED that directly targets evaporation,” Saunders said. “We expect to make MIEBO commercially available in the second half of this year.”

Dry eye disease, DED affects millions of Americans and is one of the most common ocular surface disorders.² A leading cause of DED is excessive tear evaporation, which due to an altered tear lipid layer, is often associated with the clinical signs of Meibomian gland dysfunction (MGD). An unstable tear film triggers increased ocular surface desiccation, inflammation and damage to the ocular surface.^3,4

Ophthalmology Times reached out to members of its editorial advisory board along with ophthalmologists who specialize in dry eye for their comments on what this approval would mean for their patients with dry eye.

Peter J. McDonnell, MD, director of the Wilmer Eye Institute and chief medical editor of Ophthalmology Times, noted that dry eye disease is a “special” condition because it is not a single disease but rather an assortment of pathophysiologies that result in a constellation of symptoms (burning, stinging, foreign body sensation, ocular surface staining, tear film instability, etc) that we have come to categorize as “dry eye.”

“For some patients, inflammation is the key factor, and CsA and corticosteroids have been approved for that subset,” McDonnell noted. “For others, meibomian gland dysfunction is central, and therapeutic interventions target those glands, while other patients have a deficiency of goblet cells and the mucin they generate.”

McDonnell noted that other patients diagnosed with the disease have tear hyposecretion, and there is an FDA-approved drug for stimulating aqueous tear secretion. He noted that “personalized medicine has arrived in the management of dry eye.”

“This latest approval focuses on individuals with excessive tearfilm evaporation,” he said. “As additional therapies become available to help our patients suffering from poor quality of life from dry eye, clinicians will be challenged to avoid thinking that all dry eye patients are the same, and instead segment them into the fundamental underlying pathology/ies and institute the therapy that best addresses the cause of each patient’s difficulties.”

Laura M. Periman, MD, director of Dry Eye Services and Clinical Research, Periman Eye Institute in Seattle, Washington, noted that the approval is good news for patients diagnosed with dry eye disease.

“We were a phase 3 study site and have early experience with perfluorohexyloctane,” she said. “With this approval, US doctors now have access to a first in class alkane drop as a topical ophthalmic therapeutic for the signs and symptoms of dry eye disease. “

Periman said she envisions perfluorohexyloctane ophthalmic solution being useful early and often in a variety of clinical scenarios and clinical severities (mild to severe) - as a targeted and specific strategy to immediately stabilize the tear film.

Karolinne Rocha, MD, PhD, director, Cornea & Refractive Surgery, Storm Eye Institute (Medical University of South Carolina), and a member of the Ophthalmology Times Editorial Advisory Board, also welcomed news of the FDA approval.

“NOV03 treats signs and symptoms of dry eye disease associated with meibomian gland dysfunction,” Rocha said. “This new nonaqueous drop has amphiphilic properties, due to which it forms a layer on the tear film surface to reduce tear evaporation at the ocular surface, improving the dysfunctional tear film lipid layer, a huge unmet need for our dry eye patients.”

Cynthia Matossian, MD, FACS, founder of Matossian Eye Associates and past medical director, pointed out that current lifestyles with digital devices for work, entertainment, and family connectivity wreak havoc on the tear film.

“Decreased blink rates and excessive tear evaporation fuel DED,” she said. “MIEBO, from Bausch + Lomb, has just arrived as the newest treatment option to help improve tear film homeostasis.”

Matossian noted that with the FDA approval, perfluorohexyloctane ophthalmic solution is now available for ophthalmologists and optometrists to recommend to their patients who suffer from meibomian gland disease, the major contributor to dry eye symptoms.

Ernest W. Kornmehl, MD, of Kornmehl Laser Eye Associates, Harvard and Tufts Universities and an associate medical editor for Ophthalmology Times, noted that the therapeutichas several advantages over currently available drops, including “low volume, surface tension and viscosity and are amphiphilic.”

"We will only know that this is truly a major advance once it has been used by thousands of patients under real world conditions,” Kornmehl added.

Clinical Data

The FDA approval of perfluorohexyloctane ophthalmic solution was based on results from the GOBI and MOJAVE 57-day, multi-center, randomized, double-masked, saline-controlled studies, which enrolled a total of 1217 patients with a history of DED and clinical signs of MGD,^5,6 a major cause of development and disease progression.8 An estimated 86% of people with DED have excessive tear evaporation whereby MGD is the major contributor.^7-9

In the GOBI and MOJAVE phase 3 pivotal studies, perfluorohexyloctane ophthalmic solution met both primary sign and symptom efficacy endpoints. The two primary endpoints were change from baseline at week eight (day 57 ± 2) in total corneal fluorescein staining (tCFS) and eye dryness Visual Analog Scale (VAS) score. Patients experienced relief of symptoms as early as day 15 and through day 57 with statistically significant reduction in VAS eye dryness score favoring perfluorohexyloctane ophthalmic solution observed in both studies. Additionally, at days 15 and day 57, a significant reduction in tCFS favoring perfluorohexyloctane ophthalmic solution was observed in both studies.

The most common adverse reactions experienced with perfluorohexyloctane ophthalmic solution were blurred vision (1.3-3%) and eye redness (1-3%).

References

1. Bausch + Lomb and Novaliq Announce FDA Approval of MIEBOTM (Perfluorohexyloctane Ophthalmic Solution) for the Treatment of the Signs and Symptoms of Dry Eye Disease. Bausch + Lomb Corporation. Published May 18, 2023. https://ir.bausch.com/press-releases/bausch-lomb-and-novaliq-announce-fda-approval-miebotm-perfluorohexyloctane‌

2. National Eye Institute. Dry Eye. Last updated April 8, 2022. Accessed May 2, 2023. https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/dry-eye

3. Craig JP, Nelson JD, Azar DT, et al. TFOS DEWS II report executive summary. Ocul Surf. 2017;15(4):802-812. doi:10.1016/j.jtos.2017.08.003

4. Pflugfelder SC, Paiva CS. The pathophysiology of dry eye disease: what we know and future directions for research. Ophthalmology. 2017;124(suppl 11):S4-S13. doi:10.1016/j.ophtha.2017.07.010

5. Tauber J, Berdy GJ, Wirta DL, et al. NOV03 for dry eye disease associated with meibomian gland dysfunction: Results of the Randomized Phase 3 GOBI Study. Ophthalmology. 2023;130(5):516-524. doi:10.1016/j.ophtha.2022.12.021

6. Sheppard J, Kurata F, Epitropoulos AT, et al. NOV03 for signs and symptoms of dry eye disease associated with meibomian gland dysfunction: The randomized phase 3 Mojave study. American Journal of Ophthalmology. Published online ahead of print. March 20 2023. doi:10.1016/j.ajo.2023.03.008

7. Chhadva P, Goldhardt R, Galor A. Meibomian Gland Disease: The Role of Gland Dysfunction in Dry Eye Disease. Ophthalmology. 2017;124(11S):S20-S26. doi:10.1016/j.ophtha.2017.05.031

8. Findlay, Q., & Reid, K. (2018). Dry eye disease: when to treat and when to refer. Australian prescriber, 41(5), 160-163. https://doi.org/10.18773/austprescr.2018.048

9. Lemp, MA, Crews, LA, Bron AJ. (2012). Distribution of Aqueous-Deficient and Evaporative Dry Eye in a Clinic-Based Patient Cohort: a retrospective study. Cornea, 31(5), 472-478. 2012;31(5):472-478. doi:10.109/ICO.0b013e318225415a.