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Orphan Europe Recordati S.p.A Receives 2011 Corporate Award From the National Organization for Rare Disorders (NORD)


5/18/2011 6:55:21 AM

MILAN, ITALY and PARIS--(Marketwire - May 18, 2011) -


ORPHAN EUROPE (RECORDATI GROUP) RECEIVES 2011 CORPORATE AWARD FROM THE NATIONAL ORGANIZATION FOR RARE DISORDERS (NORD) FOR THE DEVELOPMENT OF CARBAGLU® FOR THE TREATMENT OF NAGS DEFICIENCY

Recordati announces that Orphan Europe has received the 2011 Corporate Award from the National Organization for Rare Disorders (NORD). Orphan Europe was recognized for making Carbaglu® (carglumic acid) tablets available in the U.S. for the treatment of a condition known as NAGS deficiency.

The award was presented yesterday, 17 May 2011, during NORD's Annual Gala in Washington, DC by Patricia C. Richardson on behalf of NORD. "NORD salutes Orphan Europe for its commitment to the rare disease community" says Patricia Richardson, guest MC with Frank Sasinowski, Chair of the NORD Board of Directors. "We are proud to present this 2011 Corporate Award to the company".

Carbaglu® is the first specific treatment approved by the FDA for N- AcetylGlutamate Synthase (NAGS) deficiency, a rare inherited metabolic disorder resulting in high levels of ammonium in blood. NAGS deficiency is a devastating disease. Newborns presenting with symptoms caused by this condition require immediate medical care effective in lowering the high ammonium levels back to normal. Should this fail, the condition can lead to permanent damage to the brain or death (Albrecht 1998; Broere 2000, Caldovic 2002, Guffon 1995, Schubiger 1991). To Orphan Europe, it was unacceptable that a treatment available to patients in Europe was not available in the US thus depriving US patients of the benefits provided by Carbaglu®. The efforts to gain approval for Carbaglu® in the US have been rewarded by the granting of marketing approval by the FDA in March 2010.

"We are tremendously honored by NORD's acknowledgement of our work" says Marco Liguori, CEO of Orphan Europe. "Since our foundation, Orphan Europe has strived to make the lives of patients with rare diseases better by developing and bringing to market treatments for various rare diseases in Europe. It is with great pride and gratefulness that we today receive this award in honor of our efforts to expand our mission into the US market."

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. In the US, Orphan Europe is present with Carbaglu® for the treatment of N-AcetylGlutamate Synthase (NAGS) deficiency. 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. www.orphan-europe.com

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.

More information can be found athttp://www.carbaglu.net

NAGS deficiency

NAGS deficiency is an inherited metabolic disorder and it's the rarest among the Urea Cycle Disorders (UCDs) (Caldovic 2010). 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 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.

Indication

Carbaglu® (carglumic acid) is a Carbamoyl Phosphate Synthetase 1 (CPS1) activator indicated as:

-- Adjunctive therapy for the treatment of acute hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthase (NAGS).

-- Maintenance therapy for the treatment of chronic hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthase (NAGS).

Carbaglu® Important Safety Information

Carbaglu® treatment should be initiated by a physician experienced in the treatment of metabolic disorders. Any episode of acute symptomatic hyperammonemia should be treated as a life-threatening emergency.Treatment of hyperammonemia may require dialysis, preferably hemodialysis, to remove a large burden of ammonia. 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.

Management of hyperammonemia due to NAGS deficiency should be done in coordination with medical personnel experienced in metabolic disorders. Ongoing monitoring of plasma ammonia levels, neurological status, laboratory tests and clinical responses in patients receiving Carbaglu is crucial to assess patient response to treatment. Plasma ammonia levels should be maintained within normal range for age via individual dose adjustment. During acute hyperammonemic episodes, protein restriction and hypercaloric intake is recommended to block ammonia generating catabolic pathways. When plasma ammonia levels have normalized, protein intake can usually be increased with the goal of unrestricted protein intake.

The most common adverse reactions in more than 13% of patients are: infections, vomiting, abdominal pain, pyrexia, tonsilitis, anemia, ear infection, diarrhea, nasopharyngitis, and headache.

To report SUSPECTED ADVERSE REACTIONS, contact Accredo Health Group Inc. at 1-888-454-8860, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. No drug interaction studies have been performed with Carbaglu®, and human milk- feeding is not recommended. Carbaglu® tablets should not be swallowed whole or crushed. Each tablet should be dispersed in a minimum of 2.5 mL of water. Carbaglu tablets do not dissolve completely in water and undissolved particles of the tablet may remain in the mixing container. The mixing container should be rinsed with additional volumes of water and the contents swallowed immediately. USE IN OTHER FOODS AND LIQUIDS HAS NOT BEEN STUDIED CLINICALLY AND IS THEREFORE NOT RECOMMENDED. Before opening, store refrigerated at 2 to 8 oC (36 to 46 oF). After first opening of the container do not refrigerate, and do not store above 30 °C (86 °F). Keep the container tightly closed in order to protect from moisture. Write the date of opening on the tablet container. Discard one month after first opening.

Recordati, established in 1926, is a European pharmaceutical group, listed on the Italian Stock Exchange (Reuters RECI.MI, Bloomberg REC IM, ISIN IT 0003828271),with a total staff of over 2,800, dedicated to the research, development, manufacturing and marketing of pharmaceuticals. It has headquarters in Milan, Italy, operations in the main European countries, and a growing presence in the new markets of Central and Eastern Europe. A European field force of around 1,400 medical representatives promotes a wide range of innovative pharmaceuticals, both proprietary and under license, in a number of therapeutic areas including a specialized business dedicated to treatments for rare diseases. Recordati's current and growing coverage of the European pharmaceutical market makes it a partner of choice for new product licenses from companies which do not have European marketing organizations. Recordati is committed to the research and development of new drug entities within the cardiovascular and urogenital therapeutic areas and of treatments for rare diseases. Consolidated revenue for 2010 was EUR 728.1 million, operating income was EUR 154.8 million and net income was EUR 108.6 million.

Statements contained in this release, other than historical facts, are "forward- looking statements" (as such term is defined in the Private Securities Litigation Reform Act of 1995). These statements are based on currently available information, on current best estimates, and on assumptions believed to be reasonable. This information, these estimates and assumptions may prove to be incomplete or erroneous, and involve numerous risks and uncertainties, beyond the Company's control. Hence, actual results may differ materially from those expressed or implied by such forward-looking statements. All mentions and descriptions of Recordati products are intended solely as information on the general nature of the company's activities and are not intended to indicate the advisability of administering any product in any particular instance.

ORPHAN EUROPE (RECORDATI GROUP) RECEIVES 2011 CORPORATE AWARD: http://hugin.info/143644/R/1516924/453319.pdf


This announcement is distributed by Thomson Reuters on behalf of Thomson Reuters clients. The owner of this announcement warrants that: (i) the releases contained herein are protected by copyright and other applicable laws; and (ii) they are solely responsible for the content, accuracy and originality of the information contained therein.

Source: RECORDATI via Thomson Reuters ONE

[HUG#1516924]


Please refer to www.Carbaglu.net for full prescribing information
For further information please contact:
Orphan EuropeMedia Contact:
Nicole Schlautmann
Orphan Europe
+33(0) 1 47 73 86 10
Email Contact

For further information:

Recordati website: www.recordati.com

Investor Relations
Marianne Tatschke
(39)0248787393
e-mail: Email Contact



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