Drugs -- especially those that treat fatal diseases -- often entail great risks for the patient. In making its final decision on drugs, the U.S. Food and Drug Administration has to make sure the balance tips in favor of a greater benefit that makes the risk worthwhile. With that in mind, on Monday an FDA advisory panel recommended approval of Bristol-Myers Squibb's (BMY) belatacept, an experimental drug used in kidney transplants -- with at least one caveat.

Patients with chronic renal disease sometimes need a kidney transplant. But the body can reject the new organ, so patients take drugs that suppress that immune system response and allow the new kidney to function. The BMS drug in question is an investigational biologic agent, injected following renal transplantation to prevent rejection and maintain kidney function.

BMS is seeking full FDA approval of the drug, saying belatacept offers benefits over current therapies. Studies showed more patients on belatacept survived two years following a kidney transplant compared with patients taking older drugs. Patients also had lower blood pressure and better kidney function, while experiencing less toxicity associated with other rejection drugs.

The FDA's Cardiovascular and Renal Drugs Advisory Committee seemed to agree with BMS, to a degree. Members recommended approving the drug by a 13-5 vote, saying overall data supported belatacept, even though it wasn't as effective as current standard treatments such as cyclosporine. Such agents are sold by Abbott Labs (ABT) and Novartis (NVS).

Several Serious Concerns

While the panel found it important to give transplant patients and doctors more treatment options, these experts also said they would like to see additional studies performed to examine belatacept's long-term safety and effectiveness. Such studies can be done after the drug has been approved, they said, suggesting a tight patient registry, at least at first. While the FDA normally follows the recommendation of its advisory panels, it doesn't have to. The FDA's decision in the matter is expected by May 1.

The panel noted several concerns, including potentially fatal side effects in patients taking the drug. There were higher rates of severe kidney rejection in certain patients, as well as "post-transplant lymphoproliferative disorder" -- a type of lymphoma (cancer of certain immune system cells) associated with organ-transplant patients. Also, there were instances of a rare neurological disorder called "progressive multifocal leukoencephalopathy," which attacks the brain and central nervous system and is usually fatal. BMS suggests a lower dose of the drug could minimize these risks.

If the drug is approved, the Associated Press reports that Seamus Fernandez, an analyst at Leerink Swann, forecasts sales of $50 million in 2010 and $650 million by 2016. That may not be a blockbuster, but it's hardly insignificant.

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