Orphan Europe, part of the Recordati Group, is a pharmaceutical company that develops and distributes orphan drugs for the treatment of rare diseases. Whether you are a physician seeking help and support in your fight to treat a rare disease, a patient looking for information or a company looking for a partnership – Orphan Europe is waiting to hear from you.
 

 
 

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Carbaglu Advisory Committee Meeting, USA

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The 01/02/2010 at 15:48:51

The FDA requested a Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) for the review of Carbaglu which   is currently in the process of New Drug Approval (NDA) review in the USA. The EMDAC held their meeting on 13th January 2010 in which the efficacy, potential side effects and overall risk/benefit profile of Carbaglu were discussed. The EMDAC was very positive and notably the vote was unanimous for its risk/benefit profile.

This is an important step towards the NDA approval which will provide great benefit to patients with NAGS deficiency and their families.

Carbaglu® (carglumic acid) currently has marketing authorization in Europe since 2003 for the treatment of N-Acetyl Glutamate Synthase (NAGS) deficiency (more information about NAGS).

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Orphan Europe provides treatment for patients suffering from rare diseases all over the world. Read more about the different diseases here.
Orphan Europe has been developing and distributing orphan drugs since 1990 and has today an extensive product portfolio. Read more about some of the orphan medicinal products provided by Orphan Europe.
Provides high-quality, innovative science education for healthcare personnel involved in the diagnosis and management of patients affected by rare diseases.
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