Biopharmaceuticals Neurology FDA

 News Release - January 3, 2008

Orphan Drug Designation Granted to OVATION's Clobazam for the Adjunctive Treatment of a Severe Form of Childhood Epilepsy

Patients With A Rare Childhood Epilepsy May Have Potential New Treatment Option

DEERFIELD, Ill.--(HSMN NewsFeed)--OVATION Pharmaceuticals, Inc. was granted special status by the U.S. Food and Drug Administration (FDA) for clobazam as an adjunctive treatment for Lennox-Gastaut syndrome (LGS), one of the most severe forms of childhood epilepsy that frequently persists into adulthood. The FDA grants orphan drug designation to products that are intended to treat rare or “orphaned” diseases or conditions such as LGS, which is responsible for approximately three to 10 percent of all childhood epilepsies.1 For LGS patients, as with patients fighting other rare diseases, new treatments are few and far between, making this designation by the FDA of significant importance.

“LGS is a devastating and difficult to treat form of childhood epilepsy that often leads to some form of mental retardation and other behavioral problems over time,” said Dr. Tim Cunniff, Vice President, Global Regulatory Affairs, Pharmacovigilance, and Clinical Quality Assurance from OVATION Pharmaceuticals. “Clobazam is being developed to provide physicians with an additional treatment option for patients with LGS, for whom there are very few safe and effective treatments.”

Clobazam has been designated as an orphan drug by the FDA under the auspices of the Orphan Drug Act. The Act, which marks its 25th Anniversary on January 4, 2008, provides incentives to sponsors that develop therapies intended to treat orphan conditions, which are classified as any condition or disease that affects less than 200,000 patients per year in the U.S. It is estimated that there are more than 6,000 rare diseases affecting 25 million Americans.2,3

In a Phase 2 trial, clobazam significantly reduced drop seizures as compared to baseline. Drop seizures are considered the most debilitating of the LGS seizures types and can result in severe trauma to the brain and body. The majority of adverse events (AEs) observed in this trial were mild or moderate in severity. No serious AEs resulted in premature discontinuation of drug treatment. Clobazam is currently being evaluated in a late-stage clinical trial as an adjunctive treatment for LGS. For more information about the ongoing trial, please visit: http://clinicaltrials.gov/

About Lennox-Gastaut syndrome

A rare and catastrophic form of epilepsy, LGS is characterized by several seizure types and represents approximately three to 10 percent of all childhood epilepsies.1 Onset typically occurs between three and 10 years of age. Drop attacks are frequent in LGS and responsible for most injuries associated with falls. Up to 90 percent of children with LGS are affected by mental retardation and these children commonly experience behavioral and sleep disturbances as well.4

About Clobazam

Clobazam is a 1,5-benzodiazepene and has a distinctive chemical structure as compared to other currently available benzodiazepines. It was initially developed to reduce the adverse effects associated with 1,4-benzodiazepines while still maintaining efficacy. In animal models, clobazam has been shown to work both by intensifying gamma-aminobutyric acid (GABA)-mediated inhibitory effects and by increasing activity of glutamate transporters.

Clobazam is widely available with approvals in more than 100 countries for various uses in both children and adults, including the adjunctive treatment of epilepsy and anxiety. Though not currently approved for any use in the U.S., OVATION plans to seek FDA approval for clobazam as adjunctive treatment for patients with LGS. To support this development program, a global multicenter, Phase 3 clinical trial is currently underway and enrolling patients.

Clobazam is one of the key products in OVATION’s central nervous system (CNS) development pipeline. The company is advancing its near-term pipeline on other fronts as well. In July 2007, the company initiated a pivotal clinical trial to evaluate a novel intravenous formulation of carbamazepine, a widely used oral antiepileptic drug, in adult patients with epilepsy. The launch of another oral anticonvulsant for rare and refractory epilepsies is anticipated in 2008.

About OVATION Pharmaceuticals

OVATION is a fast-growing biopharmaceutical company that develops, manufactures and markets medically necessary therapies to satisfy unmet medical needs for patients with severe illnesses. Headquartered in Deerfield, Ill., with products available in more than 85 countries, OVATION is committed to having a significant impact on patients’ lives through its focus on central nervous system (CNS), hematology/oncology, and hospital-based therapies. The five new launches the company expects over the next five years will be fueled by its late-stage CNS pipeline, which is one of the most robust in the industry. OVATION has been recognized for excellence in the global pharmaceutical and biotechnology industries with the 2006 and 2007 “Pharma Company of the Year” award from Scrip magazine for small to mid-sized enterprises. More information about the company, its products and full prescribing information may be found at www.ovationpharma.com.

1. Markand ON. Lennox-Gastaut syndrome (childhood epileptic encephalopathy). J Clin Neurophysiol. 2003;20:426-441.

2. http://www.rarediseases.org/pdf/NORD_AR_2007_final.pdf

3. http://www.fda.gov/orphan/oda.htm

4. Crumrine PK. Lennox-Gastaut syndrome. J Child Neurol. 2002;17 Suppl 1:S70-75.

Source: OVATION Pharmaceuticals

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