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Posted by on Jan 27, 2015 in Foundation NewsLine |

FDA Grants Orphan Drug Designation for Arimoclomol ~ Dateline: 01/27/2015

Dear NNPDF Families and Friends,
Orphazyme today announced that the Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has granted an orphan drug designation for Orphazyme’s Arimoclomol as a new treatment for Niemann-Pick disease type C.
The US orphan drug designation complements the corresponding EMA orphan drug designation that was granted for the program in 2014.
“The recent successful completion of a €20m Series B financing round, together with the FDA and EMA orphan drug designations, establish a solid foundation for initiating our clinical studies in NP‑C with the purpose of providing arimoclomol as a safe and effective therapeutic option to patients suffering from this devastating disease as well as relief to their families,” says Dr. Carlos R. Camozzi, Chief Medical Officer of Orphazyme.
“The National Niemann-Pick Disease Foundation (NNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the NNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions ( about medical information provided on the Internet.” 

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