Regulatory Focus™ > News Articles > Pfizer's Tofacitinib Wins Regulatory Approval Ahead of PDUFA Date

Pfizer's Tofacitinib Wins Regulatory Approval Ahead of PDUFA Date

Posted 07 November 2012 | By

After a lengthy back and forth, Pfizer's potential rheumatoid arthritis blockbuster Xeljanz (tofacatinib) has won approval from the US Food and Drug Administration (FDA), the agency announced on 6 November.

Rheumatoid arthritis (RA) is a disease in which the body's immune system attacks otherwise healthy tissue, causing joint and tissue swelling and damage. Xeljanz is expected to fill a large niche market for patients with RA who did not respond to methotrexate, also used to treat RA in some patients.

The drug was subject to extensive testing, with safety and efficacy data generated from seven clinical trials. FDA said that, "In all of the trials, patients treated with Xeljanz experienced improvement in clinical response and physical functioning compared to patients treated with a placebo."

The drug will carry a Boxed Warning as a result of its safety profile, which includes an increased risk of causing serious infections, cancer and lymphoma. A number of other risks, including heightened cholesterol and decreased blood count are also present, but are not included in the Boxed Warning.

Xeljanz was also approved with a Risk Evaluation and Mitigation Strategy (REMS), which is aimed at informing patients and prescribers about the risks of prescribing Xeljanz through the use of a Medication Guide (MedGuide) and a prescriber communication plan. Xeljanz's sponsor, Pfizer, will also be required to conduct two post-market clinical trials to further assess the drug's effects on heart disease, cancer and infections.

The drug, pronounced "ZELL-JANS," will retail for slightly less than $25,000 per year, according to some reports, and stands to potentially make $2.5 billion in annual sales if initial forecasts are correct. The drug had been scheduled to receive a decision on 21 November 2012-the drug's Prescription Drug User Fee Act date-but FDA officials said they had finished their safety and efficacy review early.

The drug is approved only at the 5mg dose strength. Pfizer had also been seeking a 10mg dose strength for approval, but regulators did not approve that request.


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