Seven weeks after Ariad Pharmaceuticals Inc. was forced to halt US sales of its leukemia drug, prompting an outcry from patients and doctors, the Food and Drug Administration issued new safety measures Friday that will allow the Cambridge biotech to resume selling the medicine.
An agreement reached between the FDA and Ariad calls for a more restrictive drug label and updated information on safety risks, letting it market the powerful and expensive treatment to a narrower set of patients suffering from blood cancer. The target US market will now be only about 1,300 patients, slightly more than half the 2,500 Ariad projected before halting marketing on Nov. 1.
But because the drug, called Iclusig, will be priced above $115,000 a year per patient — slightly higher than competing leukemia therapies —- even the smaller patient population should be enough for Ariad to build a viable business, executives said. The company lost more than two-thirds of its market value and laid off 40 percent of its staff after suspending Iclusig sales.
Patients who say Iclusig combats their type of leukemia hepled convinced FDA officials to keep making Iclusig available during the suspension if their doctor had already been prescribing it. On Friday, many of them celebrated.
“I did a dance in my living room,” said Beth Galliart, a California investment firm employee who has been taking the drug in a clinical trial since 2009. “I would like to believe that feedback from patients helped the process. I think we are a reminder of what was at stake and also a reminder that many of us are on Iclusig without the serious adverse events.”
Hans Loland, a Washington state computer analyst who organized an Internet group for patients with chronic myeloid leukemia, said the FDA decision removes a lot of stress.
“For us patients, this was the best possible gift we could receive as we enter the holiday season,” he said. “Our hope that with the new recommendations and education of health care providers on risks, Iclusig can resume its position in our treatment option stack.”
Also welcoming the FDA decision was Dr. Richard M. Stone, director of the adult leukemia program at Dana-Farber Cancer Institute in Boston. He joined 22 other r specialists last month in sending a letter that called on the FDA to speed up the process allowing patients who had been buying the drug commercially to win approval to keep receiving it.
“On behalf of my patients, I’m very happy,” said Stone. “This is a very effective drug for some patients who have no other options.”
Wall Street greeted the news positively as well. Shares of Ariad shot up 16.5 percent to $6.43, gaining 91 cents in Nasdaq trading Friday.
“We are eager to once again make Iclusig available to patients,” Ariad chief executive Harvey J. Berger told analysts. “To say the least, it’s been a challenging two months.”
Under the new label, Iclusig can be used to treat patients with chronic myeloid leukemia and Philadelphia chromosome positive acute lymphoblastic leukemia, if they don’t respond to or can’t tolerate other treatments. Those patients must determine, with their doctors, whether the benefits from the drug outweigh the risks of life-threatening blood clots and heart problems that were identified in clinical studies.
The new label “puts in the hands of the prescribing physician the choice as to the utilization of Iclusig,” Berger said in an interview. “Physicians who know the patients and know the severity of the leukemia and whether the patients have preexisting conditions like heart attack or stroke can take all of that into consideration.”
Doctors could also consider reducing the dosage for some patients.
After the FDA approved the drug for US sale late last year, clinical trial data emerged showing its safety risks were greater than previously known. Those dangers were highlighted in an Oct. 31 drug safety communications from the FDA that prompted Ariad to stop selling Iclusig. Ariad executives have been in active discussions with FDA regulators since then to find a way to return the drug to market. It has remained on the market in Europe, but regulators there have required the new safety information be conveyed to doctors and patients.
The quick resolution with the FDA was “remarkable,” Berger said. “We worked very hard with the FDA to define the patient population, agree on the risk profile and bring the drug back in very short order to patients for whom it’s an appropriate or indicated therapy.”
FDA spokeswoman Stephanie Yao said patient safety is the agency’s top priority so regulators couldn’t permit Iclusig to remain on sale under its original label after increased safety risks were found. But she said the agency worked with patients, doctors, and company representatives to make sure those who needed the drug could keep taking it while the new label took shape.
“This marketing suspension brought everyone to the table to discuss the data and work to identify a patient population in which the drug’s benefit outweigh the risks,” Yao said.
Martin J. Duvall, Ariad’s executive vice president and chief commercial officer, told analysts Friday that commercial sales of Iclusig would resume in mid-January.
Berger said the sales resumption means Ariad will probably rehire dozens of salespeople and medical liaisons let go because of the suspension. But the hires would be significantly less than the 160 workers idled last month, he said. “We think we can make this a cash flow positive commercial operation with fewer people,” he said.
Ariad will decide next year whether to move forward with plans to occupy a new headquarters complex under construction in Kendall Square and, if so, how much space the company will need, Berger said.