Approval for U.S. Sale of Amgen’s Drug Is Stalled : For Kidney Patients, Relief Is in Overseas Mail

The drug comes by mail order, a clear liquid in slender ampuls, carefully packed in dry ice.

The precious cargo begins its 6,500-mile journey from a pharmacy in Switzerland, travels bySwissair to Boston’s Logan Airport and from there takes its final hop by Federal Express to a medical clinic in Pasadena. There, Warren Bacon, 72, who suffers from kidney disease, gets injections of a new biotechnology drug called erythropoietin that has helped transform his life.

His wife, Nita, said that before the three-times-a-week treatments began in February her husband “never had any energy. He’d just sit with his head in his hands most of the day”; his life was like the “living dead.”

Now, Mrs. Bacon says, “the change in this man is incredible. He has much more energy. He swims, he’s started riding his bicycle, he cooks, he works in the garden. Now he may have one bad day in six or 10.”

Valerie Buhler, 65, another Pasadena resident who has kidney disease, got her first mail-order package of EPO from West Germany in March. She is on a twice-a-week injection schedule, being treated locally by her doctor. Before, “running a few errands just tired me out,” she said. “The difference has been remarkable. I don’t sleep all afternoon. I have energy and zest for life. I’m planning to take a cruise and go to Hawaii.”

Expensive Import

The mail-order service is not cheap. Buhler pays $650 a month for her EPO supply, Bacon about $1,200. Not a penny is covered by their insurance. The drug is manufactured in the United Kingdom under license by a fledgling biotechnology company called Amgen, which has its own new EPO factory in Thousand Oaks, a mere 45 miles from where Buhler and Bacon live. But that factory might as well be in Antarctica.

The problem is that EPO can’t legally be sold in this country--at least not yet. The drug has won rave reviews for its clinical test results, even in the prestigious New England Journal of Medicine, and in February the U.S. Food and Drug Administration was all but set to approve EPO for sale. Then a messy legal squabble between Amgen and its marketing partner erupted, postponing FDA approval indefinitely.

So Buhler, Bacon and a growing number of kidney disease patients who have grown impatient are importing the drug themselves. Remarkably, it’s all perfectly legal, thanks to an obscure 30-year-old FDA policy that allows individuals to import drugs that are sold overseas so long as they are for personal use and in amounts no larger than a three-month supply.

Dr. Michael Linsey, a Pasadena nephrologist who administers EPO to Bacon and Buhler, has been impressed by how well they feel. “It’s a very good drug,” he said. Linsey has heard about the many clinical tests on EPO: “Everyone has reported exceptional results with few side effects.”

Buhler and Bacon aren’t alone. Susan Burkhart, a supervisor with a Pasadena kidney dialysis clinic, said that at a meeting last month of the California Dialysis Council, about 20 clinics said they were treating patients who bought EPO from overseas.

About 80,000 people in the United States suffer from kidney failure and need dialysis to cleanse their blood of impurities; more than 20,000 of them also need frequent blood transfusions and suffer chronic anemia.

EPO can help those 20,000 patients. The drug triggers production of red blood cells that transport oxygen throughout the body. In EPO clinical tests, 97% of the patients found that they no longer need blood transfusions. Moreover, much of their old energy returned.

However, higher blood pressure has occasionally been a side effect of EPO and patients still need dialysis treatments, so physicians must monitor their condition closely.

Denise Gilbert, an analyst with Montgomery Securities, pegs the domestic EPO market at $450 million a year by the early 1990s; other analysts say the annual EPO market could reach $2 billion.

In 1985, Amgen struck a deal with a larger company, Johnson & Johnson, to help pay for EPO research. Under the agreement, Amgen would keep domestic rights to the EPO market for kidney dialysis patients, a J&J; subsidiary would sell EPO for any other use in this country and J&J; would get all rights to the European market.

Along the way, Amgen agreed to include J&J;’s medical data when it applied for FDA approval. Unfortunately, the two partners kept squabbling over, among other things, the amount and the price of the EPO J&J; would buy from Amgen. Finally Amgen refused to file J&J;’s documents. Last winter, when the FDA was about to give Amgen approval to sell EPO, J&J; sued. In March, a federal judge in Delaware ordered Amgen to amend its FDA application and include J&J;’s data, and that put a brake on the FDA’s approval. Analyst Gilbert guesses that the FDA will approve EPO for kidney dialysis patients by June, but no one really knows how long it will take.

So some patients have made it their business to master the FDA’s obscure import policy, which came back into focus last summer when FDA Commissioner Frank Young, under pressure because of the AIDS epidemic, reaffirmed the policy.

He acted in part because of a rush of imports of dextran sulphate, a 20-year-old Japanese drug used to treat high cholesterol. When some lab tests showed that the drug can slow the AIDS virus, anxious patients in the United States began ordering the drug and soon a flood of dextran sulphate was intercepted here by confused Post Office and Customs officials.

“We allow individuals to bring in personal-use quantities of drugs available overseas, provided the drug is not on our list of things that are clearly fraudulent or dangerous,” says FDA spokesman Brad Stone.

Students of Disease

Four years ago, when Bacon was stricken with kidney disease, he lost 30 pounds in a month, and, Mrs. Bacon said, “it’s been downhill all the way.” When the EPO delays began, the Bacons turned to a friend who was traveling abroad. The friend contacted a sympathetic Swiss doctor who asked for Mr. Bacon’s medical history. Last winter, Mrs. Bacon said, “I received a wire from this pharmacy in Switzerland” which offered to ship the drug directly to a medical clinic.

Much like Bacon, Valerie Buhler has become a student of her disease. Her kidney problems were first diagnosed in 1978, and gradually her energy level dwindled. “I’d always been a very active person so it was very devastating.”

EPO is a protein produced in small amounts in the kidneys. Until recently, the problem for medical science was that the body could not produce enough EPO to correct kidney-function problems. Gene resplicing technology can now prompt the production of needed rare hormones and proteins such as EPO.

Since last summer, EPO has gone on sale in such European countries as France, West Germany and Switzerland, so Buhler got in touch with a friend at the University of Munich. Eventually, a West German pharmacy agreed to sell her the drug. When the first package arrived two months ago, she was a bit nervous about whether the drug would really help. In a week she felt the difference.

Pension Helps

But Buhler is reluctant to tell other patients about her good fortune. She manages to pay for her EPO supply with the help of her late husband’s pension, but she said many other kidney patients couldn’t afford the drug. “Most people on dialysis are on Medi-Cal and it’s still a terribly expensive thing,” she said. “They live such a limited life. I don’t want to tell them how great I feel.”

Warren Bacon, a retired furniture designer, must dig even deeper to pay for the drug. An EPO dosage varies, in part, according to the patient’s size. At 6-feet-2, Bacon needs a lot more of the drug than Buhler, who is 5-feet-2. Bacon faces a $14,000-a-year bill and is eager not only for the FDA to approve EPO and hopes Medicare will pick up most of the costs for the drug.

For now, Mrs. Bacon enjoys receiving an occasional Swiss postcard from her friendly, distant pharmacist. “Of course he’s making a profit,” she noted. But if you love your husband and his life is fuller than it has been in years, there is no choice, she said. “I’ll keep buying it until it’s released here,” she said.