Pipeline: FDA approves new drug for optic neuritis, NMOSD

Article

Fast track and orphan drug designation granted.

The U.S. Food and Drug Administration (FDA) recent approval of Enspryng (satralizumab-mwge, Genentech) makes it the third approved drug for the treatment of neuromyelitis optica spectrum disorder (NMOSD) on the market.

NMOSD is a rare autoimmune disease that affects adults who are anti-aquaporin-4 or AQP4 antibody-positive. Optic neuritis, eye pain, and vision loss occur in individuals with NMOSD whose immune system mistakenly attacks healthy optic nerve cells. The disease is estimated to affect 4,000 to 8,000 Americans.

Enspryng was demonstrated to be effective in the treatment of NMOSD in two 96-week clinical studies, according to the company.

Related: Allergan receives FDA complete response letter for wet AMD therapy

The study’s first trial group included 95 adult patients; 64 of whom had antibodies against AQP4 (anti-AQP4 positive). Enspryng reduced the number of NMOSD relapses by 74 percent during this study, compared to treatment with a placebo.

The study’s second trial group included 76 adult patients; 52 of whom were anti-AQP4 positive. Enspryng reduced the number of relapses in anti-AQP4 positive patients by 78 percent during this study.

Related: Norlase Leaf laser receives CE mark approval

Enspryng’s prescribing information includes a warning for increased infection risk, including serious and potentially fatal infections—such as potential reactivation of hepatitis B and tuberculosis. Additional warnings include elevated liver enzymes, decreased neutrophil counts, and hypersensitivity reactions.

Common side effects observed include the common cold (nasopharyngitis), headache, upper respiratory tract infection, inflammation of the lining of the stomach, rash, joint pain, extremity pain, fatigue, and nausea.

Vaccination with live-attenuated or live vaccines is not recommended during treatment with Enspryng and should be administered at least 4 weeks before treatment begins.

Enspryng demonstrated the potential to address an unmet medical need for a serious condition. It’s development and review will be expedited because it received fast track designation from the FDA. It also received orphan drug designation.

Related: GenSight Biologics reports results of Lumevoq treatment on LHON patients

Recent Videos
Walline, OD, PhD, FAAO, outlines how to predict myopia progression and delay its onset in an exclusive interview with Optometry Times.
Jeffrey Walline, OD, PhD, FAAO, overviews a presentation he gave on the Bifocal Lenses in Nearsighted Kids 2 (BLINK2) cohort study at the Collaborative Community on Ophthalmic Innovation (CCOI).
Danica Marrelli, OD, FAAO, at Glaucoma 360 discussing visual field testing for patients with glaucoma
Walline, OD, PhD, FAAO, states that the best way to learn the benefits of artificial intelligence for your practice is to stay on top of continuing education attendance.
Jeffrey Walline, OD, PhD, FAAO, gives an overview of the AAO's 2024 and what to expect in 2025 and beyond.
Dr Jamie Kuzniar discusses higher order aberrations and premium scleral lenses
Abby Gillogly Harsch, OD, FAAO, FSLS, shares a specific complex case of scleral lens fitting that she presented on at this year's GSLS.
Sherrol Reynolds, OD, FAAO, values the ophthalmic-optometric collaboration on display at the summit, running from February 14-17, 2025 in San Juan, Puerto Rico.
© 2025 MJH Life Sciences

All rights reserved.