Sanofi across Rare Disease: the full competitive set, and the 3 AXLRx reports mapping the competitive, patient-flow and payer picture — each scoped to a single asset and cited to a live source.
Pegunigalsidase now challenges Fabrazyme's two-decade lead in US Fabry disease. Two IV enzyme replacement therapies meet an oral chaperone only ~35–50% of patients can take.
Five FDA-approved Gaucher type 1 therapies (three IV enzyme replacement vs two oral substrate reduction) and eliglustat's oral first-line pivot.
Two next-generation ERTs are moving to displace alglucosidase alfa in US late-onset Pompe disease. Avalglucosidase alfa and cipaglucosidase alfa plus miglustat now define the competitive set.