SignaBlok Receives Orphan Drug Designation From FDA for a First-in-Class TREM-1 Peptide Inhibitor for the Treatment of Retinopathy of Prematurity

Key Takeaways

• The FDA granted Orphan Drug Designation to SignaBlok’s triggering receptor expressed on myeloid cells 1 peptide inhibitor for treating retinopathy of prematurity
• According to the company, the designation highlights the therapy’s potential as a first-in-class, macrophage-targeted approach
• Orphan status provides development incentives, including tax credits, fee exemptions, and potential 7-year market exclusivity upon FDA approval

SignaBlok announced that the FDA has granted Orphan Drug Designation to the company’s triggering receptor expressed on myeloid cells 1 (TREM-1) peptide inhibitor for the treatment of retinopathy of prematurity (ROP).

“Orphan Drug Designation for ROP represents a significant milestone in the development of our TREM-1 therapy for the treatment of ROP,” said Alexander B. Sigalov, PhD, president and founder of SignaBlok. “This decision by the FDA recognizes the potential for a first-in-class, macrophage-targeted TREM-1 peptide inhibitor to address a high unmet need for preterm infants suffering from ROP. We are very encouraged by favorable preclinical efficacy, pharmacokinetic, and tolerability data in clinically relevant animal models. These data will be presented at the 2026 annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) to be held in Denver, Colorado, May 3 to 7, 2026.”

The FDA's Orphan Drug Designation program provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan Drug Designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions, and 7-year marketing exclusivity upon FDA approval.

Originally published online on Eyewire+.