Category:Orphan drugs
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Lists of all drugs that have received orphan status in the United States and Europe are available from the U.S. Food and Drug Administration and the European Commission, respectively:
- FDA List of Orphan Designations and Approvals
- European Commission Register of designated Orphan Medicinal Products
Pages in category "Orphan drugs"
The following 200 pages are in this category, out of approximately 475 total. This list may not reflect recent changes.
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- Lanadelumab
- Larotrectinib
- Lecanemab
- Lenacapavir
- Leniolisib
- Lenvatinib
- Letermovir
- Lifileucel
- Lintuzumab
- Lisocabtagene maraleucel
- Lithium carbonate
- Lomitapide
- Lonafarnib
- Lonapegsomatropin
- Loncastuximab tesirine
- Lorlatinib
- Losartan
- Losmapimod
- Lovotibeglogene autotemcel
- Lumacaftor
- Lumacaftor/ivacaftor
- Lumasiran
- Lurbinectedin
- Luspatercept
- Lusutrombopag
- Lutetium (177Lu) chloride
- Lutetium (177Lu) oxodotreotide
M
- Macitentan
- Macitentan/tadalafil
- Maralixibat chloride
- Margetuximab
- Maribavir
- Mavacamten
- Mecasermin rinfabate
- Melatonin as a medication and supplement
- Melphalan
- Melphalan flufenamide
- Mepolizumab
- Mesalazine
- Metformin
- Methotrexate
- Methoxsalen
- Metreleptin
- Mifamurtide
- Migalastat
- Miglustat
- Mitapivat
- Mitomycin C
- Mitotane
- Mobocertinib
- Mogamulizumab
- Momelotinib
- Mosunetuzumab
- Motixafortide
- Moxetumomab pasudotox
- Moxidectin
N
O
P
- Pacritinib
- Pafolacianine
- Palopegteriparatide
- Palovarotene
- Panobinostat
- Paromomycin
- Pasireotide
- Patisiran
- Pazopanib
- Pegargiminase
- Pegcetacoplan
- Pegunigalsidase alfa
- Pegvaliase
- Pegzilarginase
- Pembrolizumab
- Pemigatinib
- Pemtumomab
- Perflubron
- Pevonedistat
- Pexidartinib
- Pharmaceutical Accountability Foundation
- Pimozide
- Pirfenidone
- Pirtobrutinib
- Pitolisant
- Plasminogen (medication)
- Plerixafor
- Polatuzumab vedotin
- Pomalidomide
- Posaconazole
- Pozelimab
- Pralsetinib
- Pretomanid
- Progesterone (medication)
- Protein-bound paclitaxel
- Prothrombin complex concentrate
- Pyrazinamide
R
- Ranibizumab
- Ranirestat
- Rasagiline
- Ravulizumab
- Recombinant human parathyroid hormone
- Remdesivir
- Repotrectinib
- Retifanlimab
- Retigabine
- Retinyl palmitate
- Rexin G
- Rezafungin
- Ribitol
- Rifampicin
- Rifapentine
- Rifaximin
- Rilonacept
- Rimeporide
- Rintatolimod
- Ripretinib
- Risdiplam
- Rituximab
- Romidepsin
- Romiplostim
- Ropeginterferon alfa-2b
- Rovalpituzumab tesirine
- Rozanolixizumab
- Rucaparib
- Ruxolitinib
- RVSV-ZEBOV vaccine
- RVT-802
S
- Sacituzumab govitecan
- Sacrosidase
- Sapacitabine
- Satralizumab
- Sebelipase alfa
- Selinexor
- Selpercatinib
- Selumetinib
- Setanaxib
- Setmelanotide
- Silmitasertib
- Siltuximab
- Sirolimus
- Sobetirome
- Sodium nitrite
- Sodium oxybate
- Sodium phenylbutyrate
- Sodium phenylbutyrate/ursodoxicoltaurine
- Sodium stibogluconate
- Sodium thiosulfate
- Solriamfetol
- Somapacitan
- Somatrogon
- Sorafenib
- Sotalol
- Sotatercept
- Soticlestat
- Sotorasib
- Sparsentan
- Spesolimab
- Stiripentol
- Strimvelis
- Sugemalimab
- Sutimlimab