Table 1 Approved therapies for MPSs
Name of syndrome | Deficient enzyme | Generic name | Brand name | Applicant | Approved date (FDA) |
|---|---|---|---|---|---|
MPS I | IDUA | Laronidase | Aldurazyme® | BioMarin | April 30, 2003 |
MPS II | IDS | Idursulfase | Elaprase® | Shire | July 24, 2006 |
MPS IVA | GALNS | Elosulfase alfa | Vimizim® | BioMarin | February 14, 2014 |
MPS VI | ARSB | Galsulfase | Naglazyme® | BioMarin | May 31, 2005 |
MPS VII | GUSB | Vestronidase alfa | Mepsevii® | Ultragenyx pharm | November 15, 2017 |