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zoom RSS ジェンザイム製の稀な遺伝病の治療薬の不足/米国医療事情

<<   作成日時 : 2010/04/16 21:04   >>

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 製薬工場の閉鎖のために深刻な事態に。
 バイオ企業ジェンザイムが製造する2つの稀な遺伝病の治療薬の不足により、数千人以上がひどい打撃を受けている。
 昨年夏以来続く薬剤供給問題は、製薬会社としては珍しい20年かけて築かれた高価な薬剤をめぐる数千人の患者との親密な関係がぼろぼろになってしまった。
 なぜそれほど重要な薬剤の十分な予備を持たないのか、なぜ生産上の問題の解決ができないのかと感じ、患者にとって救済者だった会社に裏切られたと感じているという。ほとんどのビジネスなら廃業しているだろうが、残念なことに患者に必要な薬剤を持っている。
 昨年6月に、会社はウイルス汚染のためボストンの主力工場を閉鎖した。生きた細胞を使って薬剤を生産するため、こうした問題は生物工学工場では起こりうるということで、会社は非難されることはなかった。
 当初、薬品不足は6〜8週間続くとしていたが、その後繰り返して期限を延長した。また、スチール、ゴム、繊維の粒子が薬のいくつかの小びんで発見された。
 再三の後退発表で、昨年の収益が45億ドルだった5大バイオ企業の一つのジェンザイムの株価が下落し、2009年第4四半期に1株当り利益は26%下落した。
 薬品は高価だが、患者はゴーシェ病のCerezymeとファブリ病のFabrazymeに依存している。両者とも稀な遺伝性の酵素欠損症である。患者は2週間ごとに酵素を静脈内に補充する。
 会社は事実上米国のすべての患者を知っている。米国内には1,500人のCerezymeユーザーと1,000人未満のFabrazymeユーザーしかいない。また、薬剤費は年間20万ドルと極めて高価である。
 ウイルス汚染が起きたときに、一時的にボストンの工場で第3の薬剤で致死性の子どもの奇病ポンペ病に対する薬剤の生産に使っていたため、CerezymeとFabrazymeの在庫が異常に少なかった。ポンペ病の薬剤によって多くの患者の命が救われたという。
 しかし、高価な代償となった。ジェンザイムは現在、Cerezymeの完全供給が5月1日、Fabrazymeが第3四半期にたぶん入手可能となるであろうと伝えている。
(書きかけ)
-----------------------------------------------------
Genzyme Drug Shortage Leaves Users Feeling Betrayed
By ANDREW POLLACK
Published: April 15, 2010
http://www.nytimes.com/2010/04/16/business/16genzyme.html

Because of a drug factory shutdown, Jeannine Lipez of Lock Haven, Pa., says she can no longer even walk across the street without getting spasms in her left leg and will probably need an operation to replace an artery.

画像Jeannine Lipez, who suffers from Fabry disease, says she can no longer even walk across the street without getting spasms in her left leg.

Carol Fink of Yountville, Calif., says lack of the medicine she needed left her constantly in pain, sapped her energy and made her thinking fuzzy.

For Dr. William Schubert, an obstetrician and gynecologist in Pocatello, Idaho, the factory shutdown may have contributed to an even worse outcome. Even as his wife and doctor raced to find doses of the drug he needed, Dr. Schubert’s heart deteriorated rapidly. He died on March 6, at the age of 63.

These people, and thousands more in this country and abroad, have been hard hit by a shortage of drugs made by the biotechnology company Genzyme to treat two rare inherited diseases.

The supply problems, which have dragged on since last summer, have frayed the close relationship ― unusual in the pharmaceutical industry ― that Genzyme had carefully built with the several thousand users of its high-price medicines over the last two decades.

Some of those patients now say they feel betrayed by the company they once viewed as their savior, wondering why Genzyme did not have a sufficient reserve of such vital drugs and how the company could have stumbled so badly in trying to fix its production problems.

“If most businesses run like this they’d be out of business,” said one patient, Mark Malone of Morris, Ill. “Unfortunately, they have the drug we need.”

Last June the company temporarily shut its main factory in Boston because of contamination from a virus. Such problems can arise in biotechnology factories, which use living cells to make drugs, and few faulted the company at the time.

But Genzyme, which initially predicted that the drug shortages would last six to eight weeks, has repeatedly backtracked on when supplies would be fully restored, as it has run into further manufacturing problems. At one point, particles of steel, rubber or fiber were found in some vials of the drugs.

The repeated setbacks have hurt the earnings and stock price of Genzyme, one of the five largest biotechnology companies, with $4.5 billion in sales last year. The company’s adjusted earnings per share fell 26 percent in the fourth quarter of 2009, though they were still up 16 percent for the year. The stock which traded above $72 early last year, closed Thursday at $52.77.

The production problems have also left the company open to a proxy challenge from the investor Carl C. Icahn. The company has also said it was likely to be fined by the Food and Drug Administration.

Beyond the financial costs, though, is the toll on people whose health depends on those two drugs: Cerezyme, for Gaucher disease, and Fabrazyme, used against Fabry disease.

Both diseases are rare inherited enzyme deficiencies that allow fatty substances to build up in the body, damaging organs. The Genzyme drugs, which are typically given intravenously every two weeks, provide the missing enzyme for each disease.

Companies that sell pills for widespread conditions like diabetes or depression rarely know the identities of the people who use their products. But Genzyme knows virtually all the patients, at least in the United States.

That is in part because there are only 1,500 Cerezyme users and fewer than 1,000 Fabrazyme users in this country. It is also because the drugs are so expensive ― about $200,000 a year. Many patients turn to a Genzyme case worker to help them secure insurance or financial aid.

After Dr. Schubert was found to have Fabry disease five years ago, he and his wife sold their house and bought a smaller one to help them afford the nearly $4,000 a month in premiums their insurer began charging them to cover the drug. “We had invested everything in that, emotionally and financially,” his widow, Janet Schubert, said.

Henri A. Termeer, chief executive of Genzyme, acknowledged that the company had let patients down. “We have this enormous humility,” he said in an interview. “We have to re-earn our standing with these patients.”

Mr. Termeer said that when the viral contamination occurred, the company had unusually low reserves of Cerezyme and Fabrazyme because it was temporarily using its Boston factory to produce a third drug, one for another rare disease, Pompe, which is often fatal to the children it afflicts.

“Many patients’ lives were saved” by the Pompe drug, Mr. Termeer said. “Given that we had never had a virus before,” he said, referring to the factory contamination, “it was probably an understandable decision. But it was a high price to pay.”

Genzyme now says full supplies of Cerezyme will be available May 1, and of Fabrazyme perhaps in the third quarter.

Genzyme executives say they do not believe that the shortage was directly responsible for Dr. Schubert’s death. And even doctors and patient advocates say that while many patients have experienced increased and sometimes intense pain and fatigue since the shortage began, more serious medical complications have been rarer.

Some doctors and patients say the shortage is more worrisome for those with Fabry disease, which can damage the heart or the kidneys. Gaucher disease can cause anemia, inability of the blood to clot, enlarged livers and spleens and, over time, bone damage.

Genzyme, in consultation with doctors and patient advocacy groups, has allowed those with more severe Gaucher disease to get a full dose of drug. But about 80 percent of the patients got no drug at all for several months late last year.

With Fabry, the company decided that there was no easy way to determine which cases were the most severe. So everyone recently has been getting only 30 percent of their usual doses. Company officials and patient advocates say that some women, who tend to have less severe cases of Fabry, have donated their doses to their sons who inherited the disease.

The consequences of the shortage might have been more severe if two companies, Shire and Protalix Biotherapeutics, had not been nearing market entries for competing enzyme therapies for Gaucher disease, while Shire was testing one for Fabry disease as well.

Starting late last summer, the Food and Drug Administration allowed those drugs to be made available free to patients before regulatory approval.

So far, it appears that as many as 15 percent of the Cerezyme users in the United States have switched to a different drug. Defections from Fabrazyme appear to have been smaller because Shire’s drug for Fabry disease, Replagal, was less readily available.

Some patients, especially those who remember what Gaucher disease was like before Genzyme introduced the first treatment two decades ago, are sticking with Cerezyme, in part out of gratitude to Genzyme.

“My life would have been so different if not for their drug,” said Linda Rubenstein of Carlisle, Mass., who said Cerezyme had allowed her to have a son and a job.

Ms. Fink, of Yountville, another longtime Cerezyme user, said she had to switch in December to Shire’s drug because of her constant pain but felt disloyal to Genzyme. She had become friendly with the company’s local patient liaisons and had to break the news to them in December when they invited her for a holiday lunch.

But some people have stayed with Cerezyme because of Genzyme’s assurances that the shortage was about to end. Ms. Lipez, whose leg arteries are clogged by Fabry, said her doctors found the paperwork to order Replagal for her too burdensome.

Ms. Schubert said she did not hear about Replagal until December. Then it took two months for her husband to get it.

Dr. Schubert had a pacemaker implanted in 1999, before he knew it was Fabry disease that was causing his heart problems. When he started on Fabrazyme five years ago, his health improved, his wife said.

When doses of Fabrazyme were cut, Dr. Schubert’s condition deteriorated rapidly, Ms. Schubert said. She said she pleaded with Genzyme for more Fabrazyme. In early February, the company made an accelerated delivery of one of the two doses Dr. Schubert had been allocated for the first quarter of the year, she said.

After Ms. Schubert heard about Replagal, she said, the Fabry specialist at the University of Utah who supervised her husband’s care tried but failed to make his university a clinical trial site for the drug. Four weeks later, their family physician in Idaho began requesting individual use of the drug for Dr. Schubert.

For various reasons, the Replagal did not arrive until nearly a month later, on Feb. 24. Dr. Schubert received an immediate infusion. But by then, his condition had deteriorated.

“He said before he died, ‘You need to tell this story; this is horrible,’ ” Ms. Schubert said. “There are just too many things that fell down.”

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