Orphan Drugs — Manufacturers, Suppliers & Exporters
PharmaTradz lists 42 Orphan Drugs products from verified GMP-certified manufacturers, suppliers and exporters worldwide, available as Tablets, Injection, Capsules, Oral Solution and more. Commonly sourced molecules include Emapalumab-lzsg, Treprostinil IV, Galsulfase IV. Buyers can compare specifications, request bulk quotes, and source with regulatory documentation (COA, GMP certificates, CTD/ACTD dossiers) for import into their market.
Available dosage forms
Tablets (14) Injection (10) Capsules (7) Oral Solution (3) Capsules. (1) Intrathecal Injection (1) Injectable Solution (1) Inhalation Solution (Nebulizer) (1)Reference brands in this category
Lenalid Bkemv Soliris Phyrago Jakafi Sutent Kyprolis Tasigna Talquetamide Pomalyst Caprelsa RilutekSend us an RFP — our team sources it from verified manufacturers and reverts with quotes.
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Agalsidase beta
Strength: 35 mg
Form: Injection
Reference Brands: Fabrazyme (USA/EU)
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Alglucosidase Alfa IV
Strength: 50 mg/vial
Form: Lyophilized powder
Reference Brands: Lumizyme®(US & EU)
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Ambrisentan tablets
Strength: 5 mg and 10 mg
Form: Tablets
Reference Brands: Letairis®(US & EU); Volibris®(EU)
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Arimoclomol
Strength: 47 mg, 62mg, 93mg
Form: Capsules
Reference Brands: MIPLYFFA (USA)
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Bosentan tablet
Strength: 62.5 mg and 125 mg
Form: Tablets
Reference Brands: Tracleer®(US & EU)
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Carfilzomib injection
Strength: 60mg/vial
Form: Injection
Reference Brands: Kyprolis
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Carglumic Acid
Strength: 200mg
Form: Dispersible Tablets
Reference Brands: Carbaglu (USA/EU)
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Dasatinib 100 mg Tablet
Strength: 100 mg, 20 mg
Form: Tablets
Reference Brands: Phyrago (USA)
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Deutivacaftor + Tezacaftor + Vanzacaftor
Strength: 10 mg + 50 mg + 125mg
Form: Tablets
Reference Brands: Alyftrek (USA)
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Doxecitine + Doxribtimine
Strength: 2g+2g
Form: Oral Solution
Reference Brands: Kygevvi (USA)
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Eculizumab 300 mg Injection
Strength: 300mg/30ml
Form: Injection
Reference Brands: Bkemv (USA), Soliris (EU/LATAM)
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Edaravone injection
Strength: 30 mg/10 ml
Form: Injectable Solution
Reference Brands: Radicava(US)
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Eliglustat capsule
Strength: 84 mg
Form: Capsules
Reference Brands: Cerdelga®(US & EU)
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Emapalumab-lzsg
Strength: 10 mg/2 mL, 50 mg/10 mL
Form: Injection
Reference Brands: Gamifant (USA/EU)
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Epoprostenol injection
Strength: 1.5 mg/via;
Form: Injection
Reference Brands: Flolan®(Us & Eu)
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Galsulfase IV
Strength: 1mg/ml
Form: Lyophilized powder for IV
Reference Brands: Vimizim®(US & EU)
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Glycerol Phenylbutyrate
Strength: 1.1 g/mL
Form: Oral Solution
Reference Brands: Ravicti (USA)
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Iloprost inahalation Solution
Strength: 10 mcg/mL
Form: Inhalation Solution (Nebulizer)
Reference Brands: Ventavis®(EU & US)
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Ivacaftor tablet
Strength: 150 mg
Form: Tablets
Reference Brands: Kalydeco®(US & EU)
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Lenalidomide 5 mg Capsule
Strength: 5mg
Form: Capsules
Reference Brands: Lenalid
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Lumacaftor + Ivacaftor tablets
Strength: 200 mg lumacaftor + 125 mg ivacaftor
Form: Tablets
Reference Brands: Orkambi®(US & EU)
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Lumasiran Sodium
Strength: 94.5 mg/0.5 mL
Form: Injection
Reference Brands: Oxlumo (USA)
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Migalastat Hydrochloride
Strength: 123 mg
Form: Capsules
Reference Brands: Galafold (USA/EU)
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Miglustat capsules
Strength: 100 mg
Form: Capsules
Reference Brands: Zavesca(US)
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Nilotinib capsules
Strength: 150 mg and 200 mg
Form: Capsules
Reference Brands: Tasigna(US & EU)
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Nitisinone tablets
Strength: 2 mg and 5 mg
Form: Tablets
Reference Brands: Orfadin®
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Nusinersen Intrathecal Injection
Strength: 12 mg/5ml
Form: Intrathecal Injection
Reference Brands: Spinraza(US)
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Palovarotene
Strength: 5 mg, 10 mg
Form: Capsules
Reference Brands: Sohonos (USA/EU)
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Pasireotide Diaspartate
Strength: 0.3 mg/mL, 0.6 mg/mL, 1.2 mg/mL, 10 mg, 20 mg, 30 mg
Form: Injection
Reference Brands: Signifor (USA/EU)
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Pomalidomide Capsules
Strength: 2 mg, 4 mg, 3 mg, and 4 mg
Form: capsules.
Reference Brands: Talquetamide(US), Pomalyst(EU)
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Riluzole tablets
Strength: 50 mg
Form: Tablets
Reference Brands: Rilutek(US)
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Romiplostim injection
Strength: 250mcg/vial, 500 mcg/vial
Form: Injection
Reference Brands: Nplate®(US & EU)
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Ruxolitinib Tablet
Strength: 5mg, 15mg
Form: Tablets
Reference Brands: Jakafi
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Sapropterin Dihydrochloride tablet
Strength: 100 mg
Form: Tablets
Reference Brands: Kuvan®(US); Kuvanon®(EU)
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Sepiapterin
Strength: 1000 mg, 250 mg
Form: Tablets / oral powder
Reference Brands: Sephience (USA/EU)
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Sunitinib Malate Tablet
Strength: 25mg, 50mg
Form: Tablets
Reference Brands: Sutent (USA)
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