Edenbridge Pharmaceuticals has selected PANTHERx Rare to distribute Yargesa (miglustat) capsules for the treatment of Type 1 Gaucher disease.

The medication represents the first oral treatment option for adults with mild to moderate disease who are unable to use enzyme replacement therapy.

The glucosylceramide synthase inhibitor Yargesa exerts its effects through the substrate reduction therapy process.

This treatment method lessens the accumulation of harmful glycosphingolipids, thereby reducing symptoms.

Gaucher disease is a rare genetic disorder characterised by a deficiency in the glucocerebrosidase enzyme, which leads to the build-up of a lipid known as glucosylceramide.

PANTHERx Rare Pharmacy CEO Rob Snyder stated: “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.”

In February 2024, Chiesi Global Rare Diseases, a Chiesi Group business unit, selected PANTHERx Rare for the distribution of Filsuvez topical gel.

Filsuvez topical gel treats wounds associated with dystrophic as well as junctional epidermolysis bullosa in adults and paediatric patients aged six months and above.