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eight patients, each of whom had been given less than six months to live at the time of surgery. Though three of the patients had to undergo a second surgery to replace a trachea made with a Nanofiber scaffold, according to Green, six of the original eight are still alive, and that the two deaths weren’t related to the transplant.
Green is encouraged by the fact that Castillo is still alive five years after her surgery with an “excellent quality of life” and that she has never had to take immunosuppressive drugs. The second patient to receive one of the engineered airways, Andemariam Beyene, had been given just two weeks to live before the surgery but is still alive, with a job and a family, two and a half years later, Green adds.
In commercializing engineered tracheas, HART wants to target patients like Beyene who have trachea cancer, as well as those who have suffered tracheal trauma or who were born without a windpipe. That’s about 6,500 patients annually worldwide, a market the company estimates at about $300 million, according to SEC filings. It would then branch out into other engineered organs.
Still, there are some big steps to complete before HART can turn this into a business.
First, there’s the regulatory challenge. HART’s engineered trachea is viewed as a “combination” product by the FDA, meaning it involves elements of both a device and, because it contains human cells, a biologic. No such products have yet been approved by the FDA, says Green, though Winston Salem, NC-based Tengion is currently running a clinical trial of a similarly engineered replacement ureter.
While seeking approval for a combination product doesn’t necessarily mean HART has to run more trials than it would be necessary a traditional medical device, it does mean that the standards for quality control are heightened. While a defibrillator might inspect, say, every 10th device, HART would have to test every single engineered trachea before it went into a patient. That’s because the cells incorporated in the organ could harbor a harmful bacteria or viruses, Green says.
So far, just three of the eight patients who have received HART’s engineered tracheas did for so as part of a clinical trial, according to Green. That trial that is being conducted in Russia. HART’s also planning to start two European trials this year, and still has to get the FDA’s consent to begin enrolling patients in a study in the U.S.
HART plans to use the results of these studies, and its compassionate use cases, to build enough of a data package to file for regulatory approval in both the U.S. and Europe. HART has said publicly that it aims to have its engineered trachea approved in Europe in 2016, and in the U.S. a year later, so its real day of reckoning is a ways away.
Should HART face that day successfully, Green estimates that it could sell each engineered trachea for between $100,000 to $200,000. And he believes HART would have a solid argument for getting the technology covered by insurance because an artificial trachea could save a life while reducing healthcare costs—say, for people with “late-stage” blockages of the trachea who are constantly getting infections and requiring hospital care.
“These patients typically cost hundreds of thousands of dollars to treat today,” he says.
Despite the regulatory, manufacturing, and reimbursement challenges ahead, Green is confident that he made the right choice in spinning out from Harvard Bio along with HART. He sees the transplants that have succeeded so far as validation that it’s more a matter of time before his new company gets where it wants to go.
“If someone had tried to hire me from Harvard Bio to run a startup biotechnology company, I probably wouldn’t have been that interested,” he says. “But because we already had human data, we basically know the product works.”