(Image credit: ©Taljat/AdobeStock)

The European Medicines Agency (EMA) granted Orphan designations to MCO-010 (sonpiretigene isteparvovec) across 5 categories of retinal dystrophies. The US Food and Drug Administration (FDA) also granted RMAT designation for MCO-010 in Stargardt disease (SD). The EMA and FDA actions suggest regulatory agency recognition of the potential of MCO-010 to address significant unmet needs.

Orphan designations with the EMA

The EMA Orphan designations cover several categories. These include non-syndromic and syndromic rod-dominant and cone-dominant dystrophies, as well as macular dystrophies. This has the potential to provide disease-agnostic regulatory paths forward for dozens of retinal conditions. In the European Union, EMA Orphan status provides opportunities for protocol assistance and up to 10 years of market exclusivity following approval.¹

RMAT designation with the FDA

The new FDA RMAT designation for SD builds on prior Orphan Drug and Fast Track designations for both retinitis pigmentosa (RP) and SD. In the United States, Orphan, Fast Track, and RMAT designations provide opportunities for accelerated development and review, flexibility in trial design, and 7 years of market exclusivity upon approval.¹

Regarding this designation, Sulagna Bhattacharya, CEO of Nanoscope, ins quoted in the company’s press release¹, saying, “Securing RMAT designation for Stargardt disease in addition to our prior FDA designations for SD and RP is a major validation for our therapies that warrant expedited development and review. Combined with 5 EMA Orphan designations, these achievements highlight the global momentum behind our MCO platform as a potential vision-restoring therapy for patients with few or no treatment options.”

What is MCO?

MCO (multi-characteristic opsin) is a one-time, in-office, intravitreal disease-agnostic therapy platform that is designed to restore vision in patients with photoreceptor degeneration, including RP, SD, and geographic atrophy (GA). This therapy works by activating highly dense bipolar retinal cells to become light sensitive and employing the remaining visual circuitry following photoreceptor death. Notably, MCO does not require genetic testing, surgical intervention, or repeat dosing, which may enable broad patient applicability within existing retina office workflows.¹

MCO clinical research

• Nanoscope Therapeutics saw positive results in the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), a rolling BLA submission to the FDA has been initiated.²
• Promising results have also been seen in the STARLIGHT Phase 2 clinical trial of MCO-010 in Stargardt disease (SD) (NCT05417126), and the company plans to initiate a Phase 3 registrational trial in 2025.¹
• A Phase 2 program for MCO in GA is expected to start by the end of 2025.¹
• Other IND-ready programs include Leber congenital amaurosis (LCA).¹

Reference:

1. Nanoscope Strengthens Global Regulatory Pathways for MCO-010 with FDA RMAT and Five EMA Orphan Designations. Nanoscope Therapeutics. News Release. September 2, 2025. Accessed September 2, 2025. https://www.prnewswire.com/news-releases/nanoscope-strengthens-global-regulatory-pathways-for-mco-010-with-fda-rmat-and-five-ema-orphan-designations-302543344.html

2. Nanoscope Therapeutics. Nanoscope Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for MCO-010, the First Gene-Agnostic Therapy to Treat Retinitis Pigmentosa. Prnewswire.com. Published July 14, 2025. Accessed July 14, 2025. https://www.prnewswire.com/news-releases/nanoscope-therapeutics-initiates-rolling-submission-of-biologics-license-application-to-fda-for-mco-010-the-first-gene-agnostic-therapy-to-treat-retinitis-pigmentosa-302503892.html