New FDA Orphan Drugs for Acute Chest Syndrome, Acute Recurrent Repetitive Seizures, Pouchitis

Jill Taylor

January 11, 2008

January 11, 2008 — The US Food and Drug Administration (FDA) has granted orphan drug designation to A-001 for the prevention of acute chest syndrome in patients with sickle cell disease, JZP-8 for the treatment of recurrent acute repetitive seizures in patients with epilepsy, and AST-120 for the treatment of pouchitis.

Orphan Drug A-001 for Acute Chest Syndrome

On November 16, the FDA granted orphan drug designation to A-001 (Anthera Pharmaceuticals, Inc) for the prevention of acute chest syndrome (ACS), the leading cause of death in patients with sickle cell disease.

ACS is a pulmonary complication of sickle cell disease that is characterized by rapidly progressive pulmonary infiltrates, tachypnea, dyspnea, hypoxemia, and chest pain. Although it is more likely to occur in young children, ACS occurring in adolescents and young adults tends to be more severe. Immediate and aggressive treatment of ACS is required to prevent possible chronic pulmonary debilitation or death.

A-001 is an inhibitor of serum secretory phospholipase A2, a potent inflammatory mediator that is elevated in ACS. The drug is currently under evaluation in a phase 2 clinical trial (Investigation of the Modulation of Phospholipase in Acute ChesT Syndrome, or IMPACTS).

Orphan Drug JZP-8 for Recurrent Acute Repetitive Seizures

On January 7, Jazz Pharmaceuticals, Inc, announced that the FDA granted orphan drug designation to its JZP-8 product candidate for the treatment of recurrent acute repetitive seizures (ARS) — clusters of seizures that appear to increase in frequency or severity over a short period of time.

ARS occurs in a small subset of patients with epilepsy, despite a regimen of antiepileptic treatment. Unresolved, ARS can progress to status epilepticus, a continuous state of seizure that requires emergency treatment.

JZP-8 is a novel formulation incorporating clonazepam, a benzodiazepam prescribed for petit mal variant, akinetic, and myoclonic seizures; petit mal seizures unresponsive to succimides; and panic disorder. It is designed for delivery by nasal spray.

Orphan Drug AST-120 for Pouchitis

On December 19, the FDA granted orphan drug designation to AST-120 (Ocera Therapeutics, Inc) for the treatment of pouchitis, the inflammation of a surgically created internal pouch for the storage of stool.

Patients with certain types of chronic bowel disease, such as ulcerative colitis or familial adenomatous polyposis, may undergo surgical treatment in which a portion of their colon is removed. To prevent the need for an external appliance to collect waste, a portion of the small intestine is used to create an internal pouch. Pouchitis occurs in an estimated 32% of patients with an ileoanal pouch, causing symptoms such as diarrhea, abdominal cramps and pain, increased stool frequency, bleeding, fever, dehydration, and joint pain.

AST-120 is an orally administered agent composed of adsorptive, spherical carbon microspheres that has been approved in Japan to delay time to dialysis and reduce uremic symptoms of patients with chronic kidney disease. It is currently being studied in a phase 3 clinical trial for the treatment of mild to moderate fistulizing Crohn's disease.


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