New FDA Orphan Drugs: CellCept, ISIS 301012, 5-HMF

Yael Waknine

June 09, 2006

June 9, 2006 — The U.S. Food and Drug Administration (FDA) has approved orphan drug status for mycophenolate mofetil in the treatment of pemphigus vulgaris; second-generation antisense drug for the treatment of patients with homozygous familial hypercholesterolemia; and a natural, non-toxic compound for the treatment of sickle-cell disease.

Mycophenolate Mofetil (CellCept) Granted Orphan Drug Status for Pemphigus Vulgaris

On June 6, the FDA granted orphan drug designation for mycophenolate mofetil ( CellCept, made by Roche Laboratories, Inc and marketed by Aspreva Pharmaceuticals Corporation) in the treatment of pemphigus vulgaris (PV).

PV is a painful skin-blistering disease for which there are currently no adequate treatment options. According to data from the International Pemphigus Foundation, the disease affects approximately 40,000 individuals worldwide. It often begins with oral lesions that become too painful to allow eating or drinking, potentially leading to malnutrition and debilitation.

The efficacy of mycophenolate for this indication is currently being evaluated in a 52-week, randomized, double-blind, placebo-controlled comparison study of 77 patients.

According to a company news release, the primary end point for the global phase 3 trial encompasses both minimal disease activity defined as no new persistent lesions, with a low steroid dose. Completion of the study is expected in 2007.

Mycophenolate capsules, tablets, and oral suspension were previously approved for use with cyclosporine and corticosteroids in the prophylaxis of organ rejection in patients receiving allogeneic renal, cardiac or hepatic transplants. An intravenous formulation is also available for short-term (14-day) use as an alternative to the oral formulations when administered within 24 hours following transplantation.

Orphan Drug ISIS 301012 for Homozygous Familial Hypercholesterolemia

The FDA approved in May orphan drug status for an investigational drug ( ISIS 301012, made by Isis Pharmaceuticals, Inc), in the treatment of patients with homozygous familial hypercholesterolemia (HoFH). Current treatment for the rare genetic disorder is inadequate, requiring many patients to undergo apheresis

According to a company news release, the second-generation antisense drug inhibits apoB-100, a protein critical to the synthesis and transport of low density lipoprotein cholesterol (LDL) and very low density lipoprotein (VLDL). Lowering cholesterol and triglyceride levels is a key component in the prevention and management of the cardiovascular disease common in HoFH patients.

The approval was based in part on data from three low-dose cohorts of a phase 2 clinical trial, showing that use of the drug as a single agent produced rapid, dose-dependent and prolonged reductions of its target, apoB-100, with concomitant reductions in LDL, VLDL, total cholesterol and triglyceride levels in patients with high cholesterol.

Results at 3 months showed that the study drug was well tolerated, and patients receiving a dose of 200-mg/week achieved a median percent reduction from baseline of 47% in apoB-100, 42% in LDL, 34% in total cholesterol and 46% in triglycerides.

According to the news release, the phase 2 trial is being continued at higher doses (300 and 400-mg/week) of the single-agent therapy. However, no drug interactions with simvastatin or ezetimibe have been noted.

Non-Toxic Natural Compound (5-HMF) Granted Orphan Drug Status for Sickle Cell Disease

On June 6, the FDA approved orphan drug status for a 5-membered heterocyclic anti-sickling compound ( 5-HMF, made by Xechem International, Inc) in the treatment of sickle cell disease.

5-HMF is a non-toxic, natural product with a high affinity for intracellular hemoglobin, allowing specific interaction with sickle cells that does not affect other body proteins.

According to a company news release, there are approximately 70 to 80,000 patients with sickle cell disease in the US, many of whom suffer unpredictable painful crises several times a year lasting a few hours to a week or more.

Reviewed by Gary D. Vogin, MD

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