Pegunigalsidase alfa is being developed as a first-line, long-term enzyme replacement therapy in adult and paediatric patients (>3 yr) diagnosed with Fabry disease (FD). FD is an inherited disease that is caused by the lack of an enzyme called alpha galactosidase A, which breaks down and removes Gb3, a complex molecule containing sugars and fats. Symptoms include pain that spreads through the body, gastrointestinal complications, headaches, impaired sweating and hearing impairment. FD is a long‐term debilitating and life-threatening disease due to recurrent episodes of severe pain not responding to painkillers.
Pegunigalsidase alfa is produced by a method known as ‘recombinant DNA technology’: it is made by cells into which a gene (DNA) has been introduced, which makes them able to produce the enzyme. The replacement enzyme has also been modified to reduce the rate at which it is removed from the body, allowing it to act for longer. Pegunigalsidase alfa was designed to increase amount of medicine in the blood and reduce the ability of a substance to provoke an immune response, thereby enhancing efficacy compared with available products. If licensed, pegunigalsidase alfa would offer an additional first-line, long -term ERT options for adult and paediatric patients (>3 yr) diagnosed with FD.
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.