Researchers conducted a 17-week study to see if a new oral drug, bitopertin, could help adults with erythropoietic protoporphyria (EPP). EPP is a rare disorder where sunlight causes severe, burning pain. The study involved 75 adults who took either a 20 mg dose, a 60 mg dose, or a placebo pill daily. The main goal was to see if the drug lowered levels of a protein in the blood called protoporphyrin-IX, which causes the light sensitivity.
By the end of the study, both doses of bitopertin significantly reduced this protein level compared to the placebo. The higher dose reduced it by nearly 50%. The study also found that people taking bitopertin had fewer painful skin reactions to light. The drug was reported to be well-tolerated with no major safety concerns identified during the trial.
It is important to be cautious about these results. This was a phase 2 trial, which means it was relatively small and only lasted about four months. The researchers noted the study was too short to know about long-term safety or benefits, and the small number of people means the findings need to be confirmed. Currently, there are no approved drugs that modify the course of EPP.
For now, this research shows bitopertin is a promising candidate that successfully lowered a key marker of the disease in a short-term study. The next steps will require much larger and longer trials to see if reducing this protein reliably leads to less pain and more time in the sun for people living with EPP.