FDA panel favors Bristol-Myers transplant drug despite higher rates of acute rejection

FDA panel back Bristol-Myers transplant drug

SILVER SPRING, Md. — Federal health advisers said Monday an organ transplant drug from Bristol-Myers Squibb Co. should be approved for patients receiving a new kidney, despite mixed results when compared with older drugs.

The Food and Drug Administration drug advisory panel voted 13-5 in favor of the company’s drug belatacept to prevent organ rejection in patients implanted with a new kidney.

FDA is not required to follow the group’s advice, though it often does.

New York-based Bristol presented its drug as a first-of-a-kind alternative to decades-old transplant drugs that cause headaches, nausea and occasionally liver toxicity.

In company studies of the drug, patients taking belatacept were more likely to survive two years following a kidney transplant than patients taking older drugs. Belatacept patients also showed improved kidney function and lower blood pressure, both key predictors of survival.

However, the FDA panelists struggled to describe the benefits of the drug, citing higher rates of severe kidney rejection compared with older drugs from Novartis and Abbott Laboratories.

“I’m not convinced they measured effectiveness adequately,” said Dr. Darren Mcguire of the University of Texas Southwestern Medical Center, who voted against approval. “I’m not willing to sacrifice a detriment to acute rejection for speculated benefits.”

The majority of the panel ultimately favored the drug as a valuable option for patients, despite its mixed results for preventing organ rejection. But nearly all panelists agreed the company should be required to conduct follow-up studies on belatacept’s long-term safety. Most of the patients in Bristol’s study were followed for just two years.

“It’s the issues we don’t yet know that worry me more than the ones we already know about,” said panel chair Dr. Emil Paganini of Critical Care Nephrology Consulting.

Along with acute organ rejection, patients taking belatacept were more likely to contract a rare neurological disease known as progressive multifocal leukoencephalopathy, which attacks the brain and central nervous system and is usually fatal.

Bristol Myers spokeswoman Laura Hortas said in a statement the company “is confident in the clinical development program that evaluated the safety and efficacy of belatacept.”

Kidney specialists have praised the drug’s ability to extend patient lifespans, though its risks could prevent it from gaining quick acceptance, according to Leerink Swann analyst Seamus Fernandez.

“They do not expect swift uptake because of the significantly higher rate of acute rejection seen with belatacept,” Fernandez stated in a recent investment note.

Assuming the drug is approved, he forecast sales of $50 million in 2010 and $650 million by 2016.

About 500,000 patients in the U.S. suffer from end-stage renal disease, or kidney failure. About 17,000 patients receive a kidney transplant each year, while the rest undergo kidney dialysis to remove waste from their bodies.

Ten years after receiving a transplant, 50 percent of patients will die, return to dialysis or require a new kidney transplant, according to medical journal research.