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:
Pages in category "Orphan drugs"
The following 200 pages are in this category, out of approximately 472 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
- 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
- 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
- Sutimlimab