New FDA Orphan Drugs for Chronic Lymphocytic Leukemia, Lennox-Gastaut Syndrome, Pediatric Crohn's Disease

Jill Taylor

January 18, 2008

January 18, 2008 — The US Food and Drug Administration (FDA) has granted orphan drug designation to oral fludarabine phosphate for the treatment of chronic lymphocytic leukemia, clobazam for the adjunctive treatment of Lennox-Gastaut syndrome, and oral beclomethasone dipropionate for the treatment of pediatric Crohn's disease.

Orphan Drug Fludarabine Phosphate for Chronic Lymphocytic Leukemia

On December 18, the FDA granted orphan drug designation to fludarabine phosphate (Xanthus Pharmaceuticals, Inc) oral tablets for the treatment of chronic lymphocytic leukemia (CLL), the most common cancer affecting the bone marrow and blood.

CLL affects an estimated 95,579 people. Although the rate of disease progression can vary widely between individual patients, most patients require treatment at diagnosis or shortly after. At present, there is no cure for CLL, and the overall goal of treatment is to maintain the health of the patient for as long as possible. Intravenous fludarabine has been available in the United States for treatment of CLL since 2003.

Fludarabine is an antimetabolite cytotoxic agent that shortens the lifespan of leukemia cells and prevents new leukemia cells from growing. According to the manufacturer, the activity and tolerability profile of oral fludarabine is similar to the intravenous formulation.

Orphan Drug Clobazam for Lennox-Gastaut Syndrome

On December 18, the FDA granted orphan drug designation to clobazam (Ovation Pharmaceuticals, Inc) for the adjunctive treatment of Lennox-Gastaut syndrome (LGS), a severe form of childhood epilepsy that is associated with impaired cognition, developmental delays, and behavioral disturbances.

Patients with LGS may experience multiple types of seizures, which frequently do not respond to a single antiepileptic treatment. Although LGS can be caused by brain malformation, perinatal asphyxia, severe head trauma, central nervous system infection, or inherited disorders, no cause can be identified in up to 35% of patients. There is currently no cure for LGS, and achieving complete seizure control is rare.

Clobazam is a 1,5-benzodiazepene initially developed to reduce the adverse effects associated with 1,4-benzodiazepine treatment. The drug has been demonstrated to intensify gamma-aminobutyric acid–mediated inhibitory effect as well as increase the activity of glutamate transporters in animal models.

Orphan Drug Beclomethasone Dipropionate for Pediatric Crohn's Disease

On January 4, DOR BioPharma, Inc, announced that the FDA granted orphan drug designation to oral beclomethasone dipropionate (BDP) for the treatment of pediatric Crohn's disease, a disorder that causes inflammation of the gastrointestinal tract.

The symptoms of Crohn's disease include abdominal pain, fever, nausea, vomiting, weight loss, and diarrhea. Although the majority of patients with Crohn's disease are diagnosed in early adulthood, approximately 30% develop symptoms before age 20. Children with Crohn's disease are at risk for growth retardation and delay of sexual maturation and are also susceptible to complications from long-term medical treatment. Patients who do not adequately respond to medication or who develop complications require surgical treatment.

BDP is a potent corticosteroid with anti-inflammatory and immunomodulating properties. It is currently approved for use by inhaler for the treatment of chronic bronchial asthma.

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