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Baltimore's Osiris Therapeutics' Stem Cell Drug Granted Orphan Drug Status By FDA

Posted on: Thursday, 22 December 2005, 21:00 CST

By Karen Buckelew

Baltimore's Osiris Therapeutics Inc. has been granted orphan drug status by federal regulators for its lead stem cell drug candidate, Prochymal.The designation gives Osiris seven years of market exclusivity for the drug, aimed at a condition called graft vs. host disease (GVHD) that afflicts bone marrow transplant recipients. It also entitles the company to tax incentives and exemption from hefty regulatory fees.Yesterday's announcement is yet another notch in Osiris' belt - the drug received Fast Track status from the Food and Drug Administration earlier this year, expediting the process for it to receive regulatory approval for the gastrointestinal form of graft vs. host disease. The company is preparing for two human clinical trials to test the drug in the treatment of the disease in both its acute form and its severe acute form. Having both orphan drug designation and Fast Track status clearly adds support to the development of Prochymal for patients suffering from life- threatening GVHD, said C

. Randal Mills, Osiris president and CEO. We also appreciate the significant financial incentives that orphan drug status provides to Osiris.The status is reserved for drugs aimed at underserved patients, those that would treat diseases afflicting less than 200,000 people per year in the United States. Prochymal is derived from adult stem cells, far less controversial a science than the embryonic stem cell research that has become a hot- button issue at the local and federal political levels. Still, Osiris' success boosts the fight for federal funding of embryonic stem cell research, which has been restricted under the Bush administration, and even for state dollars, according to advocates.Everything's exciting when you're in that field of stem cell science, said Susan O'Brien, executive director of local advocacy group Maryland Families for Stem Cell Research Inc. It shows there's such a future there. But what we don't want to get trapped into is that adult stem cells can answer all the questions out there, she added. [Opponents of public funding] use that argument to not use embryonic stem cells. But there are limitations to adult stem cells.According to the St. Jude Children's Research Hospital's Web site, GVHD occurs when, infection-fighting cells from the donor recognize the patient's body as being different or foreign. These infection-fighting cells then attack tissues in the patient's body just as if they were attacking an infection. The acute form of the disease is fatal in about 80 percent of sufferers, according to Osiris.Prochymal uses a formulation of a certain type of adult stem cell to modulate the immune system in the treatment of immunological disorders, including Crohn's disease.Treatment for graft vs. host disease currently is available, according to Dr. Richard J. Jones, a professor of oncology and medicine and director of the bone marrow transplant program at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins. But for sufferers of the severe form of the disease, current treatments - immunosuppressive drugs and corticosteroids like prednisone - don't always do the trick.When you get a severe form of it, there really isn't any drug that's been shown to be better than the old tried- and-true drugs, steroids, said Jones. But some patients get a very serious [form of] the disease, and it doesn't work. For those patients, we need something new.

Source: The Daily Record (Baltimore)

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