New FDA Orphan Drugs: KNS-760704, Colchicine, Panobinostat

Jill Taylor

October 19, 2007

October 19, 2007 — The US Food and Drug Administration (FDA) has granted orphan drug designation to KNS-760704 for the treatment of amyotrophic lateral sclerosis, colchicine for the treatment of familial Mediterranean fever and Behcet's syndrome, and panobinostat for the topical treatment of cutaneous T-cell lymphoma.

Orphan Drug KNS-760704 for Amyotrophic Lateral Sclerosis

On October 11, the FDA granted orphan drug designation to KNS-760704 (Knopp Neurosciences, Inc) for the treatment of amyotrophic lateral sclerosis (ALS), a neurological disease estimated to affect 20,000 Americans.

ALS is a rapidly progressive, fatal disease characterized by loss of muscular control resulting from motor neuron degeneration and death. Patients gradually lose strength and voluntary use of muscles, and most die of respiratory failure within 3 to 5 years of symptom onset.

The new drug is an optical enantiomer of pramipexole, a selective, high-affinity dopamine agonist. Although both KNS-760704 and pramipexole demonstrate neuroprotective properties, the dopamine receptor affinity of KNS-760704 is less than that of pramipexole, making clinical evaluation of the new drug possible over a broader dose range.

Orphan Drug Colchicine ( VitiGam) for Familial Mediterranean Fever and Behcet's Syndrome

On September 25, the FDA granted orphan drug designation to colchicine ( VitiGam, AR Scientific, Inc) for the treatment of familial Mediterranean fever and Behcet's syndrome.

Familial Mediterranean fever, an inherited disease among people of Mediterranean ancestry, is characterized by repeated attacks of fever and inflammation, commonly in the abdomen or lungs. The attacks usually begin in childhood, between the ages of 5 and 15 years.

Behcet's syndrome is characterized by inflammation of the blood vessels, causing sores and swelling distributed throughout the body. The more serious complications of the disease include meningitis, blood clots, blindness, and inflammation in the digestive system.

There is currently no known cure for either familial Mediterranean fever or Behcet's syndrome. Treatment is focused on symptom relief.

Orphan Drug Panobinostat for Cutaneous T-cell Lymphoma

On September 26, the FDA granted orphan drug designation to panobinostat ( Faridak, Novartis Pharmaceuticals Corporation) for the topical treatment of cutaneous T-cell lymphoma (CTCL).

CTCL is a general term for several cancers that of the skin, including mycosis fungoides, Sézary syndrome, lymphomatoid papulosis, cutaneous anaplastic large cell lymphoma, adult T-cell leukemia/lymphoma, peripheral T-cell lymphoma, lymphomatoid granulomatosis, granulomatous slack skin disease, and pagetoid reticulosis. These malignancies originate in the T-lymphocytes, cells of the lymph system that play a vital role in the body's immune response to infection.

The cause of CTCL is unknown. The disease is estimated to affect 5 to 10 people per 1 million.

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