FDA Boosts Nexell With ‘Orphan Drug’ Designation
By VITA REED
Shares of Nexell Therapeutics Inc., an Irvine-based drug developer, got a boost late last week after the Food and Drug Administration granted “orphan drug” status to one of the company’s experimental compounds.
The move covers Nexell’s stem-cell-derived treatment for a blood disorder known as chronic granulomatous disease. Orphan drug status is given to treatments that may offer significant benefits for patients with serious or life-threatening diseases that occur in 200,000 or fewer individuals. If regulators approve an orphan drug for sale, the maker receives seven years of U.S. marketing exclusivity.
The news sent Nexell shares to nearly 3 last week, up from 75 cents the day before the FDA news. Nexell counted a market capitalization of $53 million as of late last week.
Getting the orphan drug status makes Nexell’s drug-development program “even more attractive,” said William Albright Jr., Nexell’s chief executive. “If we are successful (in clinical trials), it will be the first approval of a stem-cell drug product.”
Nexell is sponsoring a late-stage trial of the drug under the auspices of the National Institutes of Health.
Chronic granulomatous disease occurs in about one of every 1 million people, and is four times more common in males than females. The disease is characterized by a defect that renders a type of white blood cell unable to kill certain microorganisms. Those with the disease are susceptible to recurrent, potentially fatal infections by various bacteria and fungi.
As of right now, there is no specific corrective action for the disease, according to a Web site of the Jeffrey Modell Foundation, a nonprofit research foundation that raises money for various immunodeficiency disorders. The site said, however, that bone marrow transplantation is being considered as a treatment option.
Besides chronic granulomatous disease, Nexell is looking at other applications for its stem-cell products, such as autoimmune diseases, Albright said. Nexell also is working on a potential cancer vaccine.
Nexell’s focus is on modifying or enhancing human immune function and blood-cell formation utilizing adult blood-forming cells. Albright emphasized that the company uses cells that “do not require tissue destruction.”
As for competitors, Albright said that he was not aware of any company that is seeking to do exactly what Nexell does, but he said there are some companies that are exploring various avenues of stem-cell research, including Dendreon Corp. of Seattle, Genentech Inc. of South San Francisco and Select Therapeutics Inc. of Woburn, Mass.
