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Orphan Designations by geographical area
- Ma09-Hrpe Cells
- Macitentan
- Mammalian target of rapamcyin (mTOR) inhibitor
- Manganese, dichloro [(4aR, 13aR, 17aR, 21aR)-1, 2, 3, 4, ...tetraazacycloheptadecine-kN5, kN13, kN18, kN21, kN22]
- Mannitolum
- Maribavir
- Masitinib mesilate
- Mavoglurant
- Maytansinoid-conjugated humanized monoclonal antibody against CD56
- Maytansinoid-conjugated human monoclonal antibody against mesothelin
- Mecasermin
- Mecasermin rinfabate
- Mefloquine hydrochloride
- Melarsoprol
- Melatonin
- Meloxicam
- Melphalan
- Mepolizumab
- Mercaptamine
- Mercaptopurine (oral liquid)
- Mercaptopurine (oral suspension)
- Metastable technetium 99 [99mTc] Demogastrin 2
- Methotrexate
- Methotrexate (oral liquid)
- Methoxsalen
- Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate
- Methyl O-4-O-[2-[2-[2-[2-[[N-[(1R)-1-[[4-(aminoiminomethyl)phenyl]methyl]-[-----]-D-glucopyranoside 2,6-bis(hydrogen sulfate) octasodium salt
- Methylthioninium
- Metreleptin
- Metronidazole
- Mibefradil
- Midodrine hydrochloride
- Midostaurin
- Mifamurtide
- Mifepristone
- Migalastat hydrochloride
- Miglustat
- Milatuzumab
- Milciclib maleate
- Miltefosine
- Mitotane
- Mitoxantrone
- Mixture of seven synthetic fragments consisting of p21 RAS peptides
- Mixture of two allogeneic human pancreatic cancer cell lines stably transduced with a retroviral vector encoding the murine alpha-(1,3)-galactosyltransferase gene
- Modafinil
- Modified recombinant human C-type natriuretic peptide
- Mogamulizumab
- Monarsen
- Monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E
- Moroctocog alpha
- Multilamellar microvesicle comprising phosphatidylcholine, sphingomyelin, phosphatidylethanolamine, phosphatidylserine, phosphatidylinositol and cholesterol
- Murine anti-CD22 antibody variable region fused to truncated Pseudomonas exotoxin 38
- Murine monoclonal antibody against CD26
- Murine monoclonal antibody to GD2
- Mycophenolate mofetil
