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There is a worldwide shortage of medicines to treat hemophilia, due partly to the fact that the Bayer Pharmaceuticals plant in west Berkeley has been under fire by the Food and Drug Administration for its deficient quality control procedures. 

The National Hemophilia Foundation issued a “medical advisory” on Monday that called the shortage a “crisis” and said that “given the current severe shortage, there is a real danger that (the medicines) might not be readily available for someone in a critical emergency.” 

Berkeley’s Bayer plant is its principal producer of Kogenate and Kogenate FS, drugs that can coagulate the blood of most hemophilia patients. In December 2000, the Center for Biologics Evaluation and Research, a division of the FDA, performed inspections at the Berkeley site.  

The agency found several violations of quality control standards at the plant, including failure to establish cleaning procedures for equipment and failure to establish appropriate procedures to test drugs for strength and purity. 

According to the National Hemophilia Foundation, the FDA inspection has meant that Bayer has been able to release very little product so far this year, and will not be able to do so “for the foreseeable future.” 

In addition, several batches of Kogenate FS, the company’s successor to Kogenate, have been recalled due to the fact that they were stored at improper temperatures. After Kogenate FS went on sale, it was found that one of its ingredients decomposed at room temperature, weakening the product. 

FDA spokesperson Lenore Gelb said that “the company has responded, and we are working with them.” 

The NHF estimates that the product manufactured at the Bayer facility in Berkeley accounts for 30 percent of the global supply of all types of blood coagulants. 

Hemophilia is a genetically inherited disease, in which the body is unable to produce one of the “factors” – a type of protein – that causes blood to clot. The active ingredient in Kogenate is octocog alpha, also known as “factor VIII.” It is used to treat “type A” hemophiliacs, which account for approximately 85 percent of those who suffer from the disease.  

Kogenate and Kogenate FS are genetically engineered drugs, relatively new treatments for hemophilia. Bayer first introduced the Kogenate in 1993, after buying rights to it from Genentech, Inc.  

Previously, type A hemophilia could only be treated with octocog alpha extracted directly from human blood plasma. This approach, which is still employed in some cases, carried with it a number of serious problems. The plasma, which is donated by or purchased from volunteers, is mixed into large batches before the coagulants are removed, increasing the chance that diseases such as hepatitis or HIV are passed from donor to recipient. In the 1980’s, thousands of hemophiliacs were infected by HIV after taking plasma-based coagulants. 

Val Bias, president of the Hemophilia Foundation of Northern California, was one. 

“I got hepatitis B, hepatitis C and HIV from plasma,” he said. “In the old days, these things were seen as normal side-effects of hemophilia.” 

Kogenate is made by introducing human proteins into the kidney cells of baby hamsters and growing the resultant tissue in sterile cultures. The procedure eliminates the risk of transmissible disease.  

“Because of these drugs and their success in the hemophilia community, we have children that are actually playing sports – baseball, even karate and hockey,” said Bias. “This has been a dream come true for the community.” 

But with the current shortage of the genetically engineered product, many people have to return to plasma-based remedies. Even though the risks of plasma have been greatly reduced, Bias said, the substitution is “deeply disturbing.”  

“The return to blood products is a step backward that this community is very concerned about,” he said. “To say to a parent in 2001 that they are going to move children to plasma is absolutely horrifying.” 

In addition, according to the NHF, so many patients have been forced to switch to plasma-based coagulants that supplies of that drug are becoming scarce. Hemophiliacs are being asked to postpone any elective surgery they have planned and to reduce their intake of coagulants. 

“I’m hearing from patients is that they are not going to let their kids play,” said Bias. “It’s a return to the days when hemophiliac kids were allowed to color, and that’s it.” 

Bayer Pharmaceuticals could not be reached for comment.