A local biotechnology company has gained the world’s first regulatory approval for a stem cell-based drug.
Columbia-based Osiris Therapeutics Inc. received approval last week in Canada for the use of its Prochymal drug to treat children with the potentially deadly graft-vs-host disease, or GvHD, a complication of bone marrow transplants.
“We essentially have broken through the glass ceiling with regards to the development and approval of stem cell therapies,” said C. Randal Mills, president and CEO of Osiris.
The company expects to have the drug available for sale in Canada and to file for approval in the U.S. by the end of the year, Mills said. The company filed for Canadian approval first because the U.S. required additional analysis, which the company has completed, he said.
Prochymal is administered intravenously and created from mesenchymal stem cells found in the bone marrow of healthy 18- to 30-year-old adults. One bone marrow donation produces 10,000 doses of the drug.
While the first course of treatment for GvHD is steroids, Prochymal can be a life-saving treatment, Mills said, explaining that the disease causes patients to “peel out of their skin” and “shed out their intestinal lining.”
That’s what was happening to Sami Smith, a 9-year-old from Ponchatoula, La. who was 6 years old when she was diagnosed with acute myelogenous leukemia.
Though she received a bone marrow transplant from her sister, who was a 100 percent match, Smith developed GvHD.
When steroids didn’t help, Smith entered a clinical trial for Prochymal in mid-2010, undergoing 28 rounds of stem cell treatment.
“[The doctor] basically said, ‘This is it,’ said Smith’s mother, Gabriella Smith. “Once she got those stem cells, they put her in a place where the other drugs will work for her now.”
As first-line therapy, steroids are successful in only 30 to 50 percent of patients, and the disease is fatal in up to 80 percent of cases, according to Osiris.
Health Canada’s approval was driven by a study that showed about 60 percent of patients treated with the drug demonstrated a “clinically meaningful response,” according to the company.
Though this is the first regulatory agency to approve the drug, Prochymal is available in eight countries, including the United States, under an Expanded Access Program. That program allows patients with life-threatening diseases to use experimental drugs outside of clinical trials.
Edward Tenthoff, an analyst with Piper Jaffray & Co., estimated that the market for the drug in children in Canada is about $12 million, and expanding to treatment in adults could increase that by up to three times. The U.S. market is about a $225 million market, he said.
Stem cell-based wound-healing products that are used locally, including Osiris’ own Grafix, are already on the market, “but in terms of a therapeutic drug, this is the first,” he said.
The Canadian approval could also help the company enter the market in countries that look to Canada for regulatory guidance, he said.
In a note to investors, Tenthoff estimated the company’s Canadian sales to be $1 million this year, $8 million in 2013 and $15 in 2017.
The drug is also in the late stages of clinical trials for use in treating Crohn’s disease, as well as a heart attack and type 1 diabetes treatment. Under the agreement with Health Canada, the company will perform confirmatory clinical testing of the drug.
Shares of Osiris stock rose 29 cents, or 5.5 percent, to close at $5.55 in Nasdaq trading.