Rare Disease, Orphan & Specialty Biologics
India is becoming a serious source of affordable enzyme replacement and orphan therapies.
What This Class Covers
Enzyme replacement therapy and orphan-disease biologics, offered largely as named-patient or compassionate-use supply — precisely the case an Export NOC exists for. Expect purchase-order-specific quantities and a heavier documentation load than standard generics.
Export Status Notes
Much of this trade moves as named-patient or compassionate-use supply — expect purchase-order-specific quantities and heavy documentation.
Prescription Status Notes
Not retail-dispensed.
Full Molecule List
Agalsidase alfa
Agalsidase beta
Alglucosidase alfa
Asfotase alfa
Ataluren
Betaine anhydrous
Burosumab
C1-esterase inhibitor
Carglumic acid
Chenodeoxycholic acid
Cysteamine
Deflazacort (DMD)
Eculizumab
Edaravone
Elapegademase
Eliglustat
Elosulfase alfa
Eteplersen
Hydroxocobalamin (MMA)
Icatibant
Idursulfase
Imiglucerase
Inotersen
Lanadelumab
Laronidase
Levocarnitine
Metreleptin
Migalastat
Miglustat
Nitisinone
Nusinersen
Patisiran
Pegvaliase
Ravulizumab
Riluzole
Risdiplam
Sapropterin
Sebelipase alfa
Sodium benzoate
Sodium phenylbutyrate
Tafamidis
Taliglucerase alfa
Teduglutide
Trientine
Ursodeoxycholic acid
Velaglucerase alfa
Vosoritide
Zinc acetate (Wilson disease)
No molecules match your search.
Showing 48 of 48 molecules tracked in this class. Contact us for a specific molecule not listed here.
Need a Molecule Not Listed Here?
Our manufacturing-partner network covers a broad range beyond this catalogue. Send us your requirement.