Portal Therapeutics, in partnership with GondolaBio, has achieved a milestone with the FDA granting orphan drug and fast track status to its oral therapy candidate, PORT-77. This investigational drug, according to Pharmaceutical Business Review, is designed to address X-linked protoporphyria (XLP) and erythropoietic protoporphyria (EPP), two rare genetic disorders that make everyday exposure to sunlight a painful and risky ordeal.
A New Approach for a Pressing Need
XLP and EPP are inherited conditions that cause a buildup of a substance called protoporphyrin IX (PPIX) in the body. When exposed to sunlight, people with these disorders experience severe skin pain and damage, and over time, the excess PPIX can also harm the liver. In total, more than 25,000 individuals in the US and Europe are affected, and treatment options remain extremely limited.
PORT-77 is a novel oral medication that targets the ABCG2 protein involved in the movement of PPIX out of cells. By blocking this pathway, the medicine aims to keep PPIX levels lower in the blood, potentially reducing both skin and liver complications. Currently, PORT-77 is being evaluated in a Phase II clinical trial.
Encouraging Early Data
In its earliest studies, including those with healthy volunteers, PORT-77 has shown it can quickly and significantly lower PPIX levels in the bloodstream. Notably, these effects were seen just hours after taking the drug, and no major safety concerns have emerged to date. This is a promising sign for a patient community in need of new therapeutic options.
GondolaBio’s CEO, Neil Kumar, emphasized the daily impact of these disorders, describing how the ever-present threat of skin and liver injury shapes the lives of those affected. He also highlighted the importance of finding treatments that can make a meaningful difference, as there are currently few alternatives.
Regulatory Recognition and What It Means
The FDA’s orphan drug status is reserved for treatments targeting rare diseases, offering benefits such as extended market exclusivity, waived fees, and tax incentives to encourage development. Fast track designation, meanwhile, is intended to speed up the review and approval process for therapies that show promise in treating serious conditions with few existing options.
Portal Therapeutics’ receipt of both designations will help accelerate the clinical and regulatory path for PORT-77, with the company expecting to share more detailed results from its ongoing Phase II trial soon.
Looking Ahead
With these FDA designations, Portal Therapeutics is well-placed to advance PORT-77 towards becoming a much-needed treatment for XLP and EPP. For patients whose lives are shaped by the need to avoid sunlight, this development represents a step closer to greater freedom and improved health.
