Xeljanz, approved by the FDA in 2012, is a first-in-class drug to treat rheumatoid arthritis. But is it safe?

Treatment options can seem overwhelming to people with rheumatoid arthritis (RA) or other rheumatic conditions. From pain medications to disease-modifying drugs, immunosuppressants, NSAIDs, and an ever-expanding list of biologic drugs, the number of pharmaceutical treatments may seem endless. Couple those options with natural and alternative treatments, and it can leave patients wondering if they’re making the right choices.

Now there’s a new kid on the block called tofacitinib (Xeljanz). Relatively new, that is. Xeljanz, which received Food and Drug Administration (FDA) approval in 2012, is becoming a more and more popular drug option for RA. It is poised to become a profitable one, too. It is already positioned to grab a big piece of the rheumatoid arthritis drug market, predicted to grow to $18.2 billion by 2023.

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As with any medication, the decision to use Xeljanz or not is up to the rheumatologist and their patient. Every patient is unique; every case is different.

But why is Xeljanz becoming more popular among doctors? In a statement to the press, analyst Ronnie Yoo, Ph.D., wrote, “Xeljanz will compete for patient share in two areas of the RA treatment [market]. First, with the non-TNF-alpha inhibitor biologics for patients who fail to respond to TNF-alpha inhibitors. Second, to a lesser degree, it has the potential to compete at the same line of therapy as the TNF-alpha inhibitors, in patients with an inadequate response to methotrexate.”

This means that Xeljanz is often prescribed when patients aren’t responding to other drugs that are normally given as a first line of defense against RA.

Xeljanz is an anti-JAK, or Janus kinase inhibitor, which targets a specific cellular process that is involved in the immune response and resulting inflammation in RA. Xeljanz is the first drug of its kind.

“I have been on Xeljanz for about six or seven months — absolutely the best drug I’ve tried for RA. That includes infusion drugs and injectables, and hardly any side effects,” said Theresa Ahl, of Severance, Colorado.

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Dr. Charles Pucevich of the Arthritis Clinic in Pennsylvania said that he “successfully prescribes Xeljanz for RA patients or those with psoriatic arthritis,” but noted that it isn’t yet approved to treat juvenile rheumatoid arthritis.

From a scientific standpoint, the drug seems to have good potential for success. A 2013 analysis of clinical studies showed that Xeljanz was more effective than a placebo and also more effective than the competing drug adalimumab (Humira).

In a strange, unrelated success story, researchers at Yale University have been able to use Xeljanz to cure a man with a rare disorder that caused him to lose all of his hair.

The first-in-class status of the drug offers hope to many for whom other medications have failed, but some patients have their reservations about Xeljanz.

Kevin Getchell of Alma, Michigan, said, “I’ve been taking Xeljanz for about a year. At my next rheumatology appointment, I’m going to ask to stop. I’ve had no improvement and it’s a hassle to get with my insurance company. It is only available to me from specialty pharmacies. The good thing is that the drug company gave me a co-pay debit card to cover the cost so I don’t have that hassle. The company is great in that regard — I just wish it worked for me.”

The European Medicines Agency — the European version of the FDA — rejected Xeljanz because, the organization said, studies have not shown a significant benefit for Xeljanz over and above other RA drugs. The medication also carries serious side effects. Side effects can include severe infections, liver damage, gastrointestinal bleeding, increased blood pressure and cholesterol, and an increased risk of cancer.

Heidi Schroeder, from Pittsburgh, Pennsylvania, said that she worries about the safety of Xeljanz. “My doctor won’t put me on it. He said it is too dangerous with my combination of rheumatic and autoimmune conditions.” Her worry is not uncommon: the immune-suppressing nature of many RA drugs is often a concern for patients and doctors.

All drugs carry risks, and only a doctor can decide what is right for a particular patient. Still, new, targeted drugs like Xeljanz continue to offer RA patients a variety of options to manage their disease, relieve pain, and improve their quality of life.