FDA Approves Orphan Drug Status for Type 1 Diabetes Stem Cell Therapy

Yael Waknine

May 07, 2010

May 7, 2010 — The US Food and Drug Administration (FDA) has approved orphan drug designation for a stem cell therapy (Prochymal; Osiris Therapeutics, Inc) in patients with newly diagnosed type 1 diabetes mellitus.

The product is an intravenous formulation of adult mesenchymal stem cells (MSCs) that are isolated from the bone marrow of healthy young adult donors, thereby avoiding the controversy associated with embryonic and fetal cell sources. Cell culture allows the large-scale production of thousands of doses from a single donation.

According to a company news release, MSCs are designed to provide therapeutic benefit by controlling inflammation, promoting tissue regeneration, and preventing scar formation. Preclinical studies indicate that MSCs may delay the progression of type 1 diabetes by preserving beta cell function.

Treatment safety and efficacy are currently being evaluated in a 62-patient, double-blind, placebo-controlled phase 2 study conducted at 20 medical centers across the United States. To be eligible, patients must be aged 12 to 35 years and have been diagnosed 2 to 20 weeks before study entry.

The MSC therapy previously was granted FDA orphan drug status and expanded access approval for graft vs host disease, allowing its use in patients whose condition is life-threatening.

Other potential indications under investigation include Crohn's disease, myocardial infarction, and pulmonary disease.

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