12/14/07 - Columbia-based Osiris Therapeutics, Inc. produces… (Doug Kapustin )
A Columbia-based biotechnology company said this week it received the world’s first government approval to market a stem cell drug, in Canada.
Osiris Therapeutics, founded in 1992, spent 17 years developing a stem cell therapy that offers anti-inflammatory and tissue-regeneration properties. The first treatment it has received approval for this week will help treat children who’ve received bone marrow transplants that their bodies have rejected. The condition, known as acute graft-versus-host disease, or GvHD, is fatal to 80 percent of the children who contract it, the company said.
C. Randal Mills, president and CEO of Osiris, said in a conference call Friday morning that the company has spent the past eight years navigating clinical trials and regulatory paperwork in a mission to be the first approved stem cell treatment in the world.
“During the past eight years, we have not wavered from that mission,” Mills said. “We now need a new mission.”
The two-decade path to market for Osiris’ drug, Prochymal, is par for the course in the biotechnology industry, where a new pharmaceutical is measured in multi-million dollar clinical trials and reviews that take years.
Prochymal is the first off-the-shelf stem cell drug approved for sale, and the first approved for GvHD, the company said. It derives its stem cells, it said, from the bone marrow of healthy adult donors between 18 and 30 years old.
Osiris is a small biotech company, with around 50 employees, in an industry where far larger competitors, with thousands of employees, usually grab the headlines with blockbuster drugs.
Yet Osiris is a key player in the state’s nascent stem cell therapies industry. Osiris is one of the world’s largest and most advanced stem cell firms, according to testimony provided by the leaders of the Maryland Stem Cell Research Fund this year in the General Assembly.
The taxpayer-subsidized fund doles out millions of dollars a year in grants to promote stem cell research; Osiris, however, has never received a grant from the fund, according to TEDCO.
This week, the fund said it will award $12.4 million in research grants to 40 projects led by university researchers from Johns Hopkins, University of Maryland and other institutions.
News of Osiris’s approval of its drug, Prochymal, sent the publicly traded company’s stock up more than 6 percent, to $5.60 in early morning trading.
Osiris is still going through the process of receiving approval from the Food and Drug Administration, Mills said. Prochymal has been in clinical trials and available to patients in the United States under the FDA’s “expanded access program.” About 60 percent of people suffering from GvHD are treated in the U.S., or between 75 and 100 patients, he said.
In Canada, between 100 and 150 patients deal with GvHD. Mills said he expects the drug to be available for sale in Canada later this year.
In Canada, the drug is approved for use in children in instances where steroids have failed to combat GvHD, but Mills said the Canada regulatory authority -- Health Canada -- wants Osiris to make the drug available to adults.
The drug is administered through an intravenous fluid line into a person’s body. The company said it has 48 patents connected to Prochymal.
Osiris is also testing Prochymal for treatment of other conditions, including Crohn’s, cardiac, pulmonary, diabetes and acute radiation syndrome.