Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.GondolaBio’s affiliate Portal Therapeutics has received orphan drug and fast track designations from the US Food and Drug Administration (FDA) for PORT-77, an investigational oral ABCG2 inhibitor, aimed at treating X-linked protoporphyria (XLP) and erythropoietic protoporphyria (EPP).
PORT-77, in Phase II development, aims to reduce PPIX efflux from red blood cells and hepatocytes, limiting liver damage. Credit: Testalize.me on Unsplash.
Subscribe to our email newsletter
In preclinical and Phase I studies in healthy participants, PORT-77 demonstrated a substantial reduction of plasma protoporphyrin IX (PPIX) through rapid ABCG2 inhibition.
This outcome has the potential to mitigate hepatobiliary and phototoxic complications in XLP and EPP. The reduction in PPIX levels occurred within hours post dosing, with no serious adverse events or tolerability issues seen so far.
GondolaBio co-founder and CEO Neil Kumar said: “For individuals living with protoporphyria, avoiding sunlight is a daily struggle that significantly affects their quality of life. The constant risk of skin and liver damage underscores the urgency of bringing disease-modifying treatments to this population as there remains a great unmet need.
“GondolaBio’s unique decentralised structure allows us to pursue treatments for rare diseases like EPP and XLP, and through these designations, we look forward to further collaboration with FDA as we advance this programme, with plans to report full Phase II data in the near-term.”
FDA’s orphan drug designation recognises new therapies for rare diseases impacting fewer than 200,000 people in the US, and provides incentives such as seven years’ market exclusivity post approval, fee waivers, trial tax credits and expedited pathways.
Fast track designation further accelerates the regulatory review of investigational drugs for serious conditions, where clinical data show promise in addressing unmet needs.
XLP and EPP, affecting over 25,000 individuals in the European Union and US, are genetic photodermatoses triggered by PPIX build-up, leading to painful cutaneous damage upon sunlight exposure.
PORT-77, now in Phase II development, is designed to reduce PPIX efflux from red blood cells and hepatocytes, aiming to limit liver damage and improve sunlight sensitivity for those affected by XLP and EPP.
