PANTHERx Rare has been chosen by Edenbridge Pharmaceuticals for the distribution of Yargesa (miglustat) capsules, the first oral treatment option for adults with mild to moderate Type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option.

Yargesa is a glucosylceramide synthase inhibitor that exerts its effects through a process called substrate reduction therapy. Through this process, Yargesa reduces the buildup of harmful glycosphingolipids and symptoms of the disease.

Gaucher disease is a rare, genetic lysosomal storage disorder where individuals lack the functioning glucocerebrosidase enzyme leading to the accumulation of a certain lipid known as glucosylceramide. This accumulation leads to manifestations including but not limited to an enlarged spleen or liver, hepatomegaly, anemia, thrombocytopenia, and bone pain.

"We appreciate Edenbridge Pharmaceuticals' commitment to making a difference in the lives of patients with Type 1 Gaucher disease. We are looking forward to serving the needs of the patients dealing with this illness, by streamlining the process associated with getting rare medications from the people who create them to the people who need them most, ultimately delivering better health outcomes to people living with rare diseases," said Rob Snyder, CEO of PANTHERx Rare Pharmacy.