SOURCE: Recordati

February 28, 2011 17:22 ET

Orphan Europe Selects Accredo Health Group to Distribute Carbaglu®

Orphan Europe Selects Accredo Health Group to Distribute Carbaglu® for the Treatment of Hyperammonemia due to an inherited and life-threatening Enzyme Deficiency

PARIS and MEMPHIS, TN--(Marketwire - February 28, 2011) - Orphan Europe announces that it has selected Accredo Health Group, Inc. to serve as the sole U.S. specialty pharmacy provider of Carbaglu (carglumic acid), the first FDA approved product to treat hyperammonemia due to N-AcetylGlutamate Synthase (NAGS) deficiency, a rare inherited metabolic disorder. This form of hyperammonemia - stemming from the absence of a key liver enzyme - is ideally diagnosed at birth. This condition, if untreated, could result in death or long-term, debilitating consequences to the nervous system from a build-up of ammonia in the bloodstream.

"Accredo's expertise with therapies for rare conditions made us the ideal partner to provide this life-saving therapy to patients," said Steven R. Fitzpatrick, president of Accredo, a wholly owned subsidiary of Medco Health Solutions, Inc. (NYSE: MHS). "Our commitment to delivering quality care to patients with complex medical conditions gives drug makers, such as Orphan Europe, confidence to work with us."

"This partnership will help provide the support and the access to treatment for this very rare but often devastating genetic disorder, NAGS deficiency. Patients must get treatment quickly to allow for the reduction of ammonia levels," said Giovanni Recordati, Chairman and CEO of Recordati (Italian Stock Exchange: REC), the parent company of Orphan Europe SARL. "Accredo's footprint of regional pharmacies gives them the unique ability to respond to these clinical demands."

Accredo offers an enhanced level of individualized service to patients with chronic and complex disease, including emergency delivery of Carbaglu to hospitals, clinical call center and reimbursement support services for Carbaglu patients. Patients and their physicians can call 1-888-454-8860 to obtain clinical, reimbursement and ordering information.

About Carbaglu

Carbaglu contains carglumic acid, a structural analogue of N-acetylglutamate. The biochemical and pharmacological characteristics of Carbaglu make it the specific treatment for NAGS deficiency (Caldovic 2004). It is the only currently available product indicated by the US FDA as adjunctive therapy for the treatment of acute hyperammonemia due to NAGS deficiency, and as maintenance therapy for the treatment of chronic hyperammonemia due to NAGS deficiency. Given orally, Carbaglu stimulates the first enzyme of the urea cycle, called CPS 1, triggering the urea cycle and normalizing blood ammonia concentration (Tuchman 2008). Uncontrolled hyperammonemia can rapidly result in brain injury/damage or death, and prompt use of all therapies necessary to reduce plasma ammonia levels is essential.

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NAGS deficiency

NAGS deficiency is an inherited metabolic disorder and it's the rarest among the Urea Cycle Disorders (UCDs) (Caldovic 2010). (Caldovic 2002; Caldovic 2003; Caldovic 2005). The urea cycle is an essential protein metabolic pathway.

NAGS is a mitochondrial liver enzyme that is essential for the urea cycle (Roth 2006). NAGS catalyzes the formation of N-acetylglutamate (NAG), which then acts as an activator of the first enzyme of the urea cycle, CPS 1. If NAGS is defective, NAG synthesis is impaired and there is no activation of CPS 1 to trigger the urea cycle. The activity of the urea cycle is regulated by the rate of synthesis of NAG. The gene for NAGS is located on chromosome 17.

The impairment of ammonia detoxification due to NAGS deficiency results in acute and chronic hyperammonemia, hyperglutaminemia and, eventually, hypocitrullinemia. Hyperammonemia and hyperglutaminemia are particularly toxic to the central nervous system (CNS) (Albrecht 1998; Broere 2000).

NAGS deficiency represents a serious life-threatening clinical condition (Caldovic 2002, Guffon 1995, Schubiger 1991). Patients with complete NAGS deficiency present with acute severe hyperammonemia within the first few days of life (Caldovic 2005, Nordenström 2007). The clinical course in the neonatal period may be lethal. Left untreated or insufficiently corrected, this condition leads to cerebral edema, coma, and eventually death. For those children who survive, psychomotor retardation is a frequent outcome (Schubiger 1991). Patients with partial NAGS deficiency (late-onset) can present symptoms at almost any time of life due to any stressful event such as an infection, trauma, vaccination (Kingsley 2006), surgery, pregnancy, etc.

About Orphan Europe

Orphan Europe was founded in 1990 with the aim to provide treatment for patients with unmet medical needs suffering from rare diseases. Since then it has grown its business very successfully, and is part of the Recordati Group since 2007. Orphan Europe provides nine orphan products to patients with the help of 130 medical, scientific and marketing specialists in Europe and Middle East countries. Through an extensive network of partnerships the drugs of Orphan Europe are also reaching patients in the rest of the world.

Orphan Europe has developed excellent relationships with clinicians, researchers, patient groups, health authorities and partnering companies. Orphan Europe, which has expertise in gaining European marketing approvals of orphan drugs, adapted its infrastructure to the particular requirements of very small production batches, distribution network of medicines for rare diseases.

About Accredo Health Group

Accredo Health Group, Inc., a wholly-owned subsidiary of Medco Health Solutions, Inc, is one of the nation's largest specialty pharmacies dedicated to providing an enhanced level of individualized service to patients with chronic and complex disease. Drugs dispensed by Accredo, which are often biotechnology drugs, frequently require special handling and clinical services to help promote patient safety and health.

About Medco Health Solutions

Medco Health Solutions, Inc. (NYSE: MHS) is pioneering the world's most advanced Pharmacy® and its clinical research and innovations are part of Medco making medicine smarter™ for approximately 65 million members.

With more than 20,000 employees dedicated to improving patient health and reducing costs for a wide range of public and private sector clients, and 2009 revenues of nearly $60 billion, Medco ranks 35th on the Fortune 500 list and is named among the world's most innovative, most admired and most trustworthy companies.

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Orphan Europe Selects Accredo Health Group to Distribute Carbaglu®:

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Contact Information

  • For further information please contact:

    Orphan EuropeMedia Contact:
    Nicole Schlautmann
    Orphan Europe
    +33(0) 1 47 73 86 10
    Email Contact

    Medco Health Solutions Media Contact:
    Ann Smith
    Medco Health Solutions, Inc.
    Email Contact