New FDA Orphan Drugs: 5(S)-(2'-hydroxyethoxy)-20(S)-Camptothecin, 5,7-dihydroxy-3-(4-hydroxyphenyl)-Chromen-4-One, Autologous Cultured Endothelial Cells on a Donor Human Corneal Disk

Jill Taylor

July 27, 2007

July 27, 2007 — The US Food and Drug Administration (FDA) has granted orphan drug designation for 5(S)-(2'-hydroxyethoxy)-20(S)-camptothecin for the treatment of osteosarcoma, 5,7-dihydroxy-3-(4-hydroxyphenyl)-chromen-4-one for the treatment of acute radiation syndrome, and autologous cultured endothelial cells on a donor human corneal disk for the treatment of Fuch's dystrophy, bullous keratopathy, and pseudophakic bullous keratopathy.

Orphan Drug 5(S)-(2'-hydroxyethoxy)-20(S)-Camptothecin for Osteosarcoma

On June 15, the FDA granted orphan drug designation to 5(S)-(2'-hydroxyethoxy)-20(S)-camptothecin (ClinTec International, Ltd) for the treatment of osteosarcoma, the most common type of cancer that develops in the bone.

Osteosarcoma weakens the bone, developing most commonly near the ends of the long bones, such as around the knees. The cancer can also spread beyond the bone into nearby tissues or be carried by the bloodstream to other bones or internal organs.

In the United States, approximately 900 cases of osteosarcoma are diagnosed annually. Almost half of these cases occur in patients younger than 20 years.

Orphan Drug 5,7-Dihydroxy-3-(4-Hydroxyphenyl)-Chromen-4-One (Bio 300) for Acute Radiation Syndrome

On June 18, the FDA granted orphan drug designation to 5,7-dihydroxy-3-(4-hydroxyphenyl)-chromen-4-one (Bio 300, Humanetics, Inc) for the treatment of acute radiation syndrome (ARS), a serious illness that occurs when most or all of the body is exposed to high doses of penetrating radiation.

Bio 300 was developed in response to a desire by the federal government to identify and procure medical countermeasures for ARS in case of a radiological or nuclear event. At present, no drugs are approved in the United States to prevent or treat radiation injury.

Bio 300 is a small, single molecular agent that achieves its effect through multiple factors such as stimulation of hematopoietic cell growth and differentiation, inhibition of protein tyrosine kinase-triggered apoptosis, anti-inflammatory effects, and potent antioxidant activity.

Orphan Drug Autologous Cultured Endothelial Cells on a Donor Human Corneal Disk for Corneal Disorders

On June 1, the FDA granted orphan drug designation to autologous cultured endothelial cells on a donor human corneal disk (Cellular Bioengineering, Inc) for the treatment of Fuch's dystrophy, bullous keratopathy, and pseudophakic bullous keratopathy.

Fuch's dystrophy is an inherited disorder in which the endothelial cells of the cornea slowly deteriorate, resulting in a build-up of fluid that causes swelling, blurred vision, and epithelial blisters that can erupt and cause pain. People with the disorder usually become symptomatic after the age of 50 years and can eventually become blind.

Bullous keratopathy is a swelling of the cornea. Similar to Fuch's dystrophy, it occurs most often in older people and results in a swollen, clouded cornea with blisters on the surface. Bullous keratopathy, which is seen in association with cataract surgery, is called either pseudophakic bullous keratopathy or aphakic bullous keratopathy. The risk of pseudophakic bullous keratopathy has increased in the modern era with numerous closed loop anterior chamber intraocular lenses.

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