EMA June orphans
June 20, 2012
THE European Medicines
Agency’s Committee for Orphan
Medicinal Products has adopted a
total of nine recommendations for
orphan designation, during its June
2012 meeting.
The orphans include: Metreleptin
for treatment of Barraquer-Simons
syndrome; Metreleptin for
Berardinelli-Seip syndrome;
Metreleptin for familial partial
lipodystrophy; and Metreleptin for
Lawrence syndrome.
Other orphans include:
Hexasodium phytate for treatment
of calciphylaxis, Sanifit Laboratoris;
Human apotransferrin for congenital
hypotransferrinaemia; and
Recombinant human pentraxin-2
for treatment of idiopathic
pulmonary fibrosis.
The final orphan is: (2S)-2-{[(2R)-
2-[({[3,3-dibutyl-7-(methylthio)-1,1-
dioxido-5-phenyl-2,3,4,5-
tetrahydro-1,2,5 benzothiadiazepin-
8-yl]oxy}acetyl)amino]-2-(4-
hydroxyphenyl)acetyl]amino}
butanoic acid for treatment of
progressive familial intrahepatic
cholestasis.
See www.ema.europa.eu.
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