In its old age, Bristol Myers' Orencia becomes first drug approved for preventing graft-versus-host disease

In its twilight, Bristol Myers Squibb’s Orencia is putting together an impressive second act.

The rheumatoid arthritis drug—originally approved in 2005—got a nice bump in 2017 with an approval for psoriatic arthritis.

Now comes another boost from the FDA with a nod as a preventative therapy for transplant rejection. On Wednesday, the U.S. drug regulator endorsed Orencia for the prevention of acute graft-versus-host disease (GVHD) in combination with certain immunosuppressants.

Orencia becomes the first drug approved as a preventative for GVHD. The blessing covers people 2 and older undergoing bone marrow or stem cell transplants from an unrelated donor.

GVHD occurs during a stem cell transplant when infused donor T cells, which are designed to attack cancer cells, instead take on healthy tissues and organs. The most common targets are the skin, liver and gastrointestinal tract. It occurs in 30% to 70% of transplant patients—depending on the donor and transplant type and other factors—and can be fatal.

While stem cell transplants are effective for leukemias and other hematological cancers, patients who receive transplants from unrelated and mismatched donors are at a high risk for developing acute HVGD.

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“Acute graft versus host disease can affect different parts of the body and become a serious post-transplant complication,” Richard Pazdur, M.D., director of the FDA’s oncology center of excellence, said in a release. “By potentially preventing the disease, more patients may successfully undergo bone marrow or stem cell transplantation with fewer complications.”

Orencia is an immunomodulator designed to disrupt the continuous cycle of T cell activation by binding to and inhibiting proteins involved in co-stimulation—a signaling process that induces T cells to attack.

The latest approval was based on a phase 2 trial of 186 patients that showed the addition of Orencia to standard prophylaxis (a calcineurin inhibitor plus methotrexate) led to significantly higher acute GVHD-free survival at 180 days after transplant for those in the mismatched unrelated donor arm. In the matched unrelated arm, Orencia provided numerically higher acute GVHD-free survival.

A real-world analysis provided similar results, according to BMS.

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Since its initial approval, Orencia has consistently gained use. It achieved (PDF) blockbuster status in 2012. Then in its first full year following the psoriatic arthritis green light (2018), it generated $2.71 billion in sales. Last year, sales grew to $3.16 billion and earnings reports from this year show no slowdown as it garnered $2.44 billion through the first three quarters.