Trade name Active substance Typology
Cerdelga ® Eliglustat Chemical product
Cystadane® Betaine anhydrous Chemical product
Elaprase ® Idursulfase Biological product
Glybera ® Adeno-associated Viral vector expressing lipoprotein lipase Gene/Cell therapy product
Kanuma ® Recombinant human lysosomal acid lipase (Sebelipase alfa) Biological product
Kuvan ® Sapropterin (tetrahydrobiopterin) Chemical product
Myozyme ® Alglucosidase alfa Biological product
Naglazyme ® Galsulfase Biological product
Orfadin ® Nitisinone Chemical product
Procysbi ® Cysteamine bitartrate Chemical product
Raxone ® Idebenone Chemical product
Scenesse ® Afamelanotide Chemical product
Strensiq ® Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein (Asfotase alfa) Biological product
Vimizim ® Elosulfase alfa (Recombinant human N-acetylgalactosamine-6-sulfatase) Biological product
Vpriv ® Velaglucerase Alfa Biological product
Zavesca ® Miglustat Chemical product