New FDA Orphan Drugs: Ganciclovir and Tissue Repair Cells

April 27, 2007

April 27, 2007 -- The US Food and Drug Administration (FDA) has granted orphan drug designation for ganciclovir ophthalmic gel in the treatment of acute herpetic keratitis and tissue repair cells in the treatment of dilated cardiomyopathy.

Orphan Drug Ganciclovir for Treatment of Acute Herpetic Keratitis

On April 16, the FDA granted orphan drug designation to ganciclovir ophthalmic gel (Sirion Therapeutics, Inc) for the treatment of acute herpetic keratitis, a herpes simplex virus that is reported to affect approximately 50,000 people annually in the United States.

With orphan drug designation, ganciclovir is the first topical ophthalmic antiviral treatment launched in more than 2 decades. The drug inhibits viral DNA synthesis as a result of phosphorylation to a substrate that competitively inhibits the binding of deoxyguanosine triphosphate to DNA polymerase. Sirion claims that the safety profile of ganciclovir is superior to that of trifluridine, the most common topical ophthalmic antiviral currently prescribed for herpes simplex keratitis.

Sirion obtained rights to the drug in January from Laboratoires Thea, which markets the drug in Europe under the brand name Virgan. Under the agreement, Sirion will be allowed to market the drug in the United States using the Virgan trade name.

Orphan Drug Tissue Repair Cells for Treatment of Dilated Cardiomyopathy

On February 1, the FDA granted orphan drug designation for tissue repair cells (TRCs; Aastrom Biosciences, Inc) for use in the treatment of dilated cardiomyopathy, a group of disorders in which the ventricles enlarge but are not able to pump an adequate supply of blood, resulting in heart failure.

Dilated cardiomyopathy affects an estimated 120,000 to 150,000 people in the United States and is characterized by a high mortality rate. To date, the only effective long-term treatment for patients with end-stage disease is heart transplantation.

Research suggests that high doses of stem cells and progenitor cells may slow or reverse disease progression in the hearts of patients with dilated cardiomyopathy. TRCs, which contain a large number of stem cells and progenitor cells derived from a small sample of the patient's bone marrow, are intended to induce heart tissue regeneration and may delay or eliminate the need for heart transplantation.


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