Biotech Values

[DewDiligence]

More on the Tofacitinib endorsement…

http://www.bloomberg.com/news/2012-05-09/pfizer-s-arthritis-pill-backed-by-u-s-fda-advisory-panel.html

›Pfizer’s Arthritis Pill Backed by U.S. FDA Advisory Panel

By Drew Armstrong - May 9, 2012 4:10 PM ET

Pfizer Inc. (PFE)’s experimental pill to treat rheumatoid arthritis, one of the drugmaker’s leading therapy candidates, should be approved by U.S. regulators, an advisory panel said.

Pfizer, the world’s largest drugmaker, is seeking to sell the pill, tofacitinib, for patients who don’t respond to other rheumatoid arthritis treatments. A panel of advisers to the Food and Drug Administration recommended in an 8-2 vote today in Silver Spring, Maryland, that the agency clear the drug for sale. The FDA isn’t required to follow the panel’s advice.

Tofacitinib is one of three experimental drugs from New York-based Pfizer that this year will receive an approval decision or report key data. If cleared for sale, the arthritis pill may generate $1.5 billion annually by 2020, John Boris, an analyst with Citigroup Inc. in New York, said in a May 7 note. [I think $1.5B is on the low side, even if the label is somewhat restrictive. RA biologics generate about $25B annually, so Tofacitinib doesn’t need to garner a big market share to greatly exceed Boris’ $1.5B forecast. (Perhaps Boris was talking about the US market, specifically.)]

While Pfizer’s pill has safety concerns, they are similar to other rheumatoid arthritis drugs on the market, said David Blumenthal, a professor of medicine at Case Western Reserve University and a member of the panel.

“This is as good as is possible at this stage in the game,” Blumenthal said at today’s panel meeting.

Autoimmune Disease

Rheumatoid arthritis is an autoimmune disease in which the body attacks itself. It causes swelling and damage in the joints, which can make basic tasks such as walking or holding items painful. The condition is treated with anti-inflammatory pills like aspirin, or drugs that attack the disease directly by modifying the immune system such as Amgen Inc. (AMGN)’s Enbrel and Abbott Laboratories’ (ABT) Humira.

About 1.3 million people in the U.S. had the disease in 2009, according to the National Institutes of Health. Early detection can help slow or stop its progression.

Pfizer rose 0.1 percent to $22.45 at the close in New York. Abbott declined 2.2 percent to $61.23 and Amgen fell 1.5 percent to $69.62.

“Pfizer appreciated the robust discussion regarding tofacitinib,” Kristen Neese, a company spokeswoman, said in an e-mail after the vote. “The clinical trial results demonstrated a favorable benefit-risk profile and support the approval of tofacitinib. We look forward to continuing our dialogue with the FDA.”

Dose Questions

The panel also voted 7-2 that the pill was safe, and 10-0 that it was effective. Questions will now turn to how broad of an indication Pfizer can get from the FDA, and whether the agency will approve the proposed 5-milligram and 10-milligram doses. The FDA is slated to decide on the drug by Aug. 21.

Panel members said they wanted to see more data on the drug’s safety profile, and asked that Pfizer conduct follow-up studies once the treatment goes on the market [naturally].

“I’m a little concerned about the data showing increasing risks,” said Lenore Buckley, a panel member and professor at the Virginia Commonwealth University School of Medicine in Richmond.

Buckley said she was concerned that infections and malignancies in patients on the drug seemed to be connected to the length of time they stayed on the medicine. “The 5-milligram dose looks better, but I’m not sure it would look better at five or 10 years,” she said.

Pfizer argued that both doses were effective and should be approved. “The committee feels some concern about the possible toxicities,” Buckley said. “I don’t think anybody on the committee has spoken out that the 10-milligram dose shouldn’t be a possibility.”

Staff Report

In a May 7 report, FDA staff said they had “serious safety concerns” about tofacitinib. Rheumatoid arthritis treatments that affect the immune system can have side effects including increased infections and cancers. Regulators also consider whether the medicines increase cardiovascular problems and kidney toxicity.

The panel also voted 8-2 that the drug didn’t stop the progression of the disease, measured by scans of patients’ joints. Panel members said that was a difficult measure, though, and one that can’t always be relied on to assess a treatment’s efficacy.

“We’re going to be setting ourselves up for difficulties by setting the bar a little bit too high,” said Leslie Crofford, chief of rheumatology at the University of Kentucky College of Medicine in Lexington, Kentucky, and a panel member.‹