Key Takeaways
- U.S. Food and Drug Administration (FDA) authorized EYLEA (aflibercept, Regeneron, Bayer) injection as the first pharmacological treatment for Retinopathy of Prematurity (ROP)
- ROP is one of the leading causes of childhood blindness globally
- ROP may occur in babies born prematurely when new, abnormal blood vessels grow in the retina, sometimes leading to retinal detachment that can lead to vision loss
- The availability of an FDA approved medication for ROP represents an important alternative for preterm infants and their parents
FDA APPROVAL
On February 8, 2023, the U.S. Food and Drug Administration (FDA) authorized EYLEA (aflibercept, Regeneron, Bayer) injection as the first pharmacological treatment for Retinopathy of Prematurity (ROP). The National Eye Institute describes ROP as an eye disease that may occur in babies born prematurely or who weigh less than three pounds at birth. ROP comes about when new, abnormal blood vessels grow in the retina, called retinal neovascularization, or NV. These abnormal blood vessels can grow in the wrong direction and can pull the retina up off the back of the eye, a form of retinal detachment that can lead to vision loss.1
POTENTIAL IMPACT
ROP is one of the leading causes of childhood blindness globally. As of November 2022, the WHO estimated that 15 million babies are born preterm annually, putting some at risk for ROP. Until the recent FDA approval, the only treatment available for ROP2 was laser photocoagulation. Laser photocoagulation can be a complex and lengthy process (1 to 1.5 hours) whereas an intravitreal injection may be performed at the bedside in as little as 5 minutes. For fragile preterm infants, the length of time under either sedation or general anesthesia is a potential issue.3
“The availability of an FDA approved medication for ROP represents an important alternative for preterm infants and their parents,” said Dr. Ronald Danis, a board-certified ophthalmologist with over 30 years of experience as a practicing medical retina specialist, as well as extensive involvement in the conduct and design of clinical trials. “Any additional treatment options that might preserve the vision of preterm infants is exciting news,” Dr. Danis added.
PHASE III TRIALS: BUTTERFLEYE AND FIREFLYE
The FDA approval is based on data from two randomized global Phase 3 trials – FIREFLEYE (N=113) and BUTTERFLEYE (N=120) – examining EYLEA 0.4 mg versus laser photocoagulation in infants with ROP. Approximately 80% of EYLEA-treated infants achieved an absence of active ROP and unfavorable structural outcomes at 52 weeks of age, which is better than would have been expected with no treatment. No new EYLEA safety signals were observed in either trial and AEs in both trials were consistent with infant prematurity or to the injection procedure, and with the AEs in similar ROP trials.4
Clinical trials of this nature are highly complex and require careful execution to obtain data to support FDA approval. For example, color fundus photographs using wide-field digital retinal photography will need to be taken at numerous timepoints over the course of an extensive study period. The procedure is made even more challenging when working with a hand-held camera with preterm infants.
A detailed protocol for color fundus photographs image acquisition and transmission needs to be provided to the sites. All images will need to be submitted to a central reading center to confirm the ROP staging or resolution, as well as the analysis of efficacy data. A specific grading grid for ROP needs to be developed and applied.
MERIT CENTRAL READING SERVICES
MERIT is an innovative, global clinical trial endpoint services provider. We partner with CROs, pharmaceutical, and biotech companies to deliver reliable Reading Center Services in global clinical trials. Together our work advances and accelerates the improvement of therapeutic options for patients worldwide.
MERIT’s comprehensive Reading Center Services include:
MEDICAL EXPERTISE. MERIT delivers medical expertise for study design insights such as protocol design and consultation as well as masked image reads and adjudication.
CLINICAL OPERATIONS. Clinical operations services comprise development of study-specific documents (e.g. Imaging Charters and procedure manuals), site technician and equipment certification, and project and site management.
TECHNOLOGY PLATFORM. MERIT’s comprehensive cloud-based software platform, EXCELSIOR™, built specifically for supporting image data collection and management for clinical trials, offers the latest web and imaging technologies for data standardization, analysis, reading, and reporting.
QUALITY ASSURANCE. Quality Assurance at MERIT forms the cornerstone for our successful management of clinical trial data. MERIT’s Quality Management System (QMS) is ISO 13485 certified. We prioritize data privacy; MERIT is GDPR compliant in both our processes and with our proprietary EXCELSIOR software. EXCELSIOR is a 510(k) cleared medical device for ophthalmic and radiological indications that is HIPAA compliant and meets all 21 CFR Part 11 requirements for electronic records and signatures.