Feb. 21, 2012 at 9:01 AM ET
Ovarian cancer patients whose treatment was interrupted or even stopped entirely because they couldn’t get supplies of the critical chemotherapy drug Doxil applauded news early Tuesday that federal health officials have found a solution to the shortage.
Food and Drug Administration officials said they have arranged to import temporarily a replacement drug called Lipodox from an Indian manufacturer, Sun Pharma Global.
"That's great. I'm very glad," said Diane Nathanson, 62, of Richmond, Va., who was diagnosed with ovarian cancer in 2006.
“It’s been such a struggle," she said. "Even when I got it, it’s like, ‘Yeah, I got it, but there’s thousands more who didn’t.’”
Nathanson, a retired chef and librarian, had her Doxil infusions postponed twice during recent treatments. On Monday, the drug was available for her, but she learned it was no longer working; a marker for her tumor progression had risen.
“Who knows if it would have worked if I’d had the drug all along?” Nathanson said.
Availability of the new drug is expected to end the Doxil crisis, FDA Commissioner Margaret Hamburg said in a statement released Tuesday.
"Through the collaborative work of FDA, industry and other stakeholders, patients and families waiting for these products or anxious about their availability should now be able to get the medications they need," Hamburg said.
This is the second significant drug shortage resolution that the FDA has announced in a week. Last week, officials said they had eased a dire shortage of methotrexate, a chemotherapy drug used to treat childhood leukemia. Ohio-based Bedford Laboratories agreed to release limited supplies of the drug produced before affiliated Ben Venue Laboratories shut down because of manufacturing problems last fall.
On Tuesday, the FDA also confirmed the agency has granted approval to APP Pharmaceuticals to produce a preservative-free version of the generic drug, the kind most needed to treat acute lymphoblastic leukemia, or A.L.L.
In addition, the agency said it has helped manufacturer Hospira to expedite release of an additional 31,000 vials of preservative-free methotrexate, enough for more than one month's worth of demand. FDA officials are also working with other manufacturers to increase production.
That should help ease the worries of Sara Stuckey of Lincoln, Ill., who was told there was only a week's worth of methotrexate left to treat her 6-year-old son, Nate, who was diagnosed in 2009 with A.L.L.
"It's hard enough to hear your child has cancer, but to hear that the treatment that is successfully working is no longer available is devastating," said Stuckey, who addressed an FDA briefing Tuesday. "My husband I pray that the recommended drug to fight his cancer will be available."
The Doxil move resolves a shortage that has made the drug virtually unavailable since June because of manufacturing problems. The drug is also used to treat multiple myeloma and AIDS-related Kaposi sarcoma.
FDA's Lipodox deal is a temporary, limited arrangement with Sun Pharma Global FZE and its distributor, Caraco Pharmaceutical Laboratories Ltd. The agency has authority to import foreign drugs on a limited basis in rare cases of a shortage of critical drugs.
FDA officials said that they had previously inspected the Indian firm, which already exports the drug to other companies.
The arrangement was first reported by USA Today on Monday.
Patients who need Doxil have been placed on waiting lists, had treatments deferred or have been forced to turn to other, perhaps less effective, regimens as a result of the short supply. Restoring access could be life-saving, said Maggie Heim, 58, of Hermosa Beach, Calif., who posted word of the deal on her Facebook page late Monday.
“Oh, that’s so excellent,” said Heim, a lawyer who has collected more than 800 signatures demanding that the drug’s supplier, Janssen Products LP, restore the scarce medication. “So I can throw my petition away, I hope?”
Sun Pharmaceuticals Industries Ltd. is based in Mumbai, India, and has a rapidly growing share of the global drug market, according to the firm’s website. Lipodox is a version of doxorubicin hydrochloride liposome injection, which can be used alone or in combination with other drugs to treat cancer.
Doxil and methotrexate were among 287 drugs in shortage in the United States, according to the University of Utah’s Drug Information Service, which has been tracking the problem.
Shortages have been mounting in recent years, up from about 74 in 2005, according to the Utah program.
The escalating problem has caught the attention of Congress, but bills requiring prompt notification of shortages are languishing., advocates say. Last October, President Barack Obama issued an executive order led to a rule that now requires drug makers to report promptly shortages of critical drugs. On Tuesday, the FDA also issued draft guidance to drugmakers on detailed requirements for both voluntary and mandatory notification of issues that could disrupt the drug supply.
Since fall, the FDA has used new information to intervene early with manufacturers to avert shortages of drugs. In 2011, the agency’s efforts avoided 195 shortages, said Erin Fox, who runs the University of Utah drug tracking program. Before the methotrexate and Doxil successes, the FDA already had avoided 18 shortages by the end of January this year.
For patients like Heim, who was diagnosed with stage 3 ovarian cancer in December 2009, the FDA’s effort to replace Doxil is reassuring after months of inaction.
“I’m not sure they do have the tools they need, but someone’s being proactive,” she said. “Having Doxil in the arsenal again is good.”