Taliglucerase alfa is a plant cell expressed recombinant form of glucocerebrosidase produced in transformed carrot root cells. It is intended as a substitute enzyme replacement therapy (ERT) for the first line treatment of patients with Type 1 Gaucher disease. Taliglucerase alfa is administered by intravenous (IV) infusion at a starting dose range of 30U/kg to 60U/kg of body weight, given over 1 to 2 hours once every 2 weeks. Dosage may be adjusted depending on the individual patient.
Leriglitazone is expected to work in patients with ALD by activating receptors called ‘PPAR gamma’, found in the mitochondria, which are components within cells that generate energy. Leriglitazone will improve the ALD pathogenic cascade by improving mitochondrial dysfunction, reducing oxidative stress and neuroinflammation, promoting demyelination and halting axonal degeneration. Hence, leriglitazone is expected to protect cells from damage and slow the progression of the disease. If licensed, leriglitazone would offer the first drug treatment option for adult ALD patients with AMN who currently have no effective therapies available.