The first new type of drug in more than a decade for keeping transplanted kidneys functioning won approval from federal regulators on Wednesday.
The Food and Drug Administration approved Nulojix, developed by Bristol-Myers Squibb Co., for preventing rejection of a transplanted kidney in adult patients, despite concerns about serious possible side effects. It was approved for use along with other medicines that suppress the immune system, including corticosteroids.
Without immune suppressants, a patient's body would reject the new kidney as a foreign object and attack it. If other drugs can't counteract that rejection, the kidney could stop its crucial job of filtering toxins from the blood, and the patient would have to undergo hours-long dialysis three times a week.
Even with the best care, about half of patients either die or have their new kidney stop working in roughly 8 to 10 years, said Dr. Brian Daniels, senior vice president for global development and medical affairs at New York-based Bristol-Myers.
"This is the first meaningful alternative in over a decade for patients, and one that provides potential for long-term survival" of transplanted kidneys, Daniels told The Associated Press in an interview.
Besides having a new mechanism of action, Daniels said Nulojix kept kidneys functioning better over three years in the company's two large, final-stage studies, compared to the standard treatment, cyclosporine. He said in a small number of patients getting Nulojix in a separate study and followed for up to eight years, kidney function had yet to decline significantly.
Nulojix, given through 30-minute intravenous infusions once a month for life, works by inhibiting the activation of T cells, which are key to triggering an immune system response to a perceived invader.
Bristol-Myers said it expects to launch the drug in early July. So far, it is not disclosing the expected price.
Nulojix, also known by the chemical name belatacept, is an alternative to some older immune suppressants, which cause headaches and nausea and, occasionally, kidney toxicity.
The approval comes 3 1/2 months after a panel of FDA advisers voted 13-5 to approve Nulojix despite concerns about higher rates of severe kidney rejection than older drugs have.
The drug carries a boxed warning, the most serious type, that it can increase risk of a type of cancer in which white blood cells grow out of control after an organ transplant, a condition called post-transplant lymphoproliferative disorder.
There's a second boxed warning that Nulojix, as well as other immune suppressants, increases risk of other cancers and serious infections.
Nulojix can also cause other side effects. Common reactions in studies that included more than 1,200 patients included anemia, constipation, kidney and bladder infections, and swollen legs, ankles or feet.
In addition, all transplant patients are at risk of infection, so they should not get live vaccines. Those taking Nulojix also should limit time spent in sunlight because of the risk of skin cancer.
More than 89,000 patients in the United States are waiting for a kidney transplant, according to the Organ Procurement and Transplantation Network.
(This version to kidney toxicity in 9th paragraph. Corrects to 13-5 in 10th paragraph)