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OrphanDrug
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OrphanDrug covers global rare drug access intelligence for 193 countries.

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Drug Database

126 entries
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Orphan Drugs Only
Mipomersen
Orphan Cold ChainFDA Approved
mitapivat
Orphan10dFDA Approved (2022), EMA Approved (2022)
Nitisinone
Orphan21dFDA Approved
NovoSeven
Orphan Cold ChainFDA Approved
Nusinersen
Orphan Cold Chain7dUnregistered in India
Octreotide
Orphan Cold Chain10dFDA Approved
Olipudase alfa
Orphan Cold Chain14dFDA Approved
Onasemnogene abeparvovec
Orphan Cold Chain14dNot registered in India
Palovarotene
Orphan14dFDA Approved
Patisiran
Orphan Cold ChainFDA Approved
Pyrimethamine
OrphanFDA Approved
Ravulizumab
Orphan Cold ChainFDA Approved
Ribavirin
Orphan7dFDA Approved
Risdiplam
Orphan7dUnregistered in India
rituximab
Orphan Cold Chain14dFDA Approved
Romidepsin
Orphan Cold Chain7dFDA Approved
Romiplostim
Orphan Cold Chain7dFDA Approved
ruxolitinib
Orphan7dFDA Approved
Sacrosidase
OrphanFDA Approved
selumetinib
Orphan14dFDA Approved, EMA Approved
sirolimus
Orphan5dFDA Approved, EMA Approved
Sodium Phenylbutyrate
OrphanFDA Approved
Somatrogon-ghla
Orphan Cold ChainFDA Approved
Somatropin
Orphan Cold Chain14dFDA Approved
Tafamidis
Orphan10dFDA Approved
temozolomide
Orphan7dFDA Approved, EMA Approved
Temsirolimus
Orphan Cold ChainFDA Approved
Tolvaptan
Orphan14dFDA Approved
trabectedin
Orphan Cold Chain21dFDA Approved
Trametinib
OrphanFDA Approved
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