LogicBio Therapeutics announced that the U.S. FDA has placed a clinical hold on the Investigational New Drug (IND) submission for LB-001 for the treatment of methylmalonic acidemia (MMA) pending the resolution of certain clinical and nonclinical questions. LogicBio...
Sanofi reported positive results from two phase 3 trials of olipudase alfa, an investigational recombinant human acid sphingomyelinase, for the treatment of acid sphingomyelinase deficiency (ASMD), also known as Nieman-Pick disease, in adult and pediatric patients....
Akcea Therapeutics along with its parent company, Ionis Pharmaceuticals, announced positive topline results from the Phase 2 study of AKCEA-ANGPTL3-LRx in patients with hypertriglyceridemia, type 2 diabetes and non-alcoholic fatty liver disease (NAFLD). The study met...
Acasti Pharma announced topline results from the phase 3 trial, TRILOGY 1, which was designed to determine the efficacy of CaPre compared to placebo in lowering fasting triglyceride (TG) levels in patients with severe hypertriglyceridemia. Despite positive results in...
Allena Pharmaceuticals announced positive topline results from URIROX-1, a Phase 3 pivotal trial evaluating reloxaliase in patients with enteric hyperoxaluria (EH). The primary endpoint was achieved, showing a statistically significant reduction in urinary oxalate in...
Clinuvel announced the U.S FDA has approved Scenesse (afamelanotide) to increase pain-free light exposure in adult patients with a history of phototoxic reactions (damage to skin) from erythropoietic protoporphyria (EPP). With this approval, Scenesse is the first...
