FELIQS, a multinational clinical-stage biopharmaceutical firm headquartered in Japan, has announced that its leading asset, FLQ-101, has received Fast Track designation from the U.S. Food and Drug Administration (FDA). This groundbreaking small molecule is designed to prevent retinopathy of prematurity (ROP), a severe eye disorder impacting premature neonates. FELIQS is set to initiate its Phase 1b/2 trial, named the tROPhy-1 study, across both the U.S. and Japan by the first quarter of 2025.

The FDA’s Fast Track program is specifically tailored to streamline the development and review process for drugs addressing serious conditions with significant unmet medical needs. This designation for FLQ-101 signifies its potential to address these gaps in neonatal care.

A first-in-class preventive solution

FLQ-101 is a daily oral or intravenous formulation that aims to enhance natural retinal vascularization while safeguarding against inflammation and abnormal blood vessel growth. In 2024, FLQ-101 had already achieved Orphan Drug designation from the FDA, a critical acknowledgment of its potential in treating rare diseases. Additionally, FELIQS is on schedule to submit an Investigational New Drug (IND) application for FLQ-104, a candidate for intermediate dry age-related macular degeneration (AMD), by the second half of 2025.

Key perspectives from leadership

Dr. Ken-ichiro (Nobu) Kuninobu, Co-founder and CEO at FELIQS, highlighted that with no approved preventive treatments for ROP currently available, the FDA’s Fast Track designation is a significant milestone for the company. He noted that this status not only accelerates FLQ-101’s review process but also enhances access to FDA feedback, potentially shortening development timelines and bolstering the likelihood of clinical success. Dr. Kuninobu emphasized FELIQS’ dedication to aiding vulnerable groups, specifically extremely premature neonates and elderly patients facing dry AMD.

An urgent need for innovation in ROP treatment

Retinopathy of prematurity is a developmental disorder marked by abnormal retinal blood vessel growth in premature infants. Statistics from 2019 indicate that ROP affected approximately 27,000 neonates in the U.S., maintaining its position as a leading cause of childhood blindness. Current treatments, including laser photocoagulation and anti-VEGF therapies, are reactive rather than preventive, necessitate anesthesia, and pose risks such as retinal detachment and potential intraocular infections. These methods also rely heavily on early detection and can lead to long-term complications.

  FELIQS, FLQ-101, FLQ-104, Japan, Ken-ichiro Kuninobu, retinopathy of prematurity, tROPhy-1 study, U.S. FDA