The USFDA has approved an injectable form of lumasiran as the first drug for treating primary hyperoxaluria (PH), a rare genetic disorder. The condition is caused due to excess production of oxalate in the body. The drug has received the designations of orphan drug and breakthrough therapy. Primary hyperoxaluria affects one in every 58,000 people worldwide. Primary hyperoxaluria type 1 is the most common and severe type of PH.
One to three individuals per million in North America and Europe are affected by PH. Patients suffering from PH experience kidney damage leading to kidney failure. Disease progression may result in oxalate deposition in other organs including the heart, bones, and eyes. The approval of lumasiran for PH is based on two studies as follows: Study parameters Study 1 Study 2 Study details Randomized, placebo-controlled Randomized Participant details Patients aged <…
