FDA recommends new Lupus drug for approval!

[from WSJ:]

A Food and Drug Administration panel of outside experts recommended Tuesday that the agency approve lupus drug Benlysta, developed by Human Genome Sciences Inc. and GlaxoSmithKline PLC.
The 13-to-2 vote increases the likelihood of the drug getting to patients with the autoimmune disease, making it the first new treatment in more than 50 years. The panel also voted that the data surrounding the drug demonstrated its effectiveness and its safety.

Lupus has historically been a difficult development area for drug makers, partly because of the varied nature of its symptoms make it hard to prove a treatment is effective in a large-scale clinical trial. Attempts to develop new treatments for the condition have created a long list of failures.
Benlysta is important for both companies as it one of the most important new drugs in Glaxo's pipeline and Human Genome Sciences has no products on the market.
Leerink Swann & Co. analysts project that global sales of Benlysta could exceed $5 billion in 2020. The drug's success in two large studies, called Bliss, has produced consistent speculation that Glaxo, or another large drug maker, would acquire Human Genome.

Lupus occurs when the body attacks itself, causing inflammation and tissue damage virtually anywhere in the body, making it both difficult to diagnose and treat. It affects about 1.5 million people nationally, according to the Lupus Foundation of America.

Benlysta is designed to help relieve lupus symptoms that can include joint pain and swelling resembling severe arthritis as well as rashes and inflammation of the kidneys. It blocks a protein required for the development of certain immune system cells thought to play a key role in lupus.

If Benlysta is approved, its label will likely include strong warnings, as many drugs for rheumatoid arthritis already do. After voting on the approval, many panel members urged that the label of the drug should note that the drug hasn't been tested in certain populations, including those with severe kidney disease.

On Tuesday, researchers who worked with the companies presented data from large clinical trials that they said showed the drug was effective.

After failing earlier testing, the companies worked with the FDA to design the trials with a never-used combination of several disease-activity measures, including the drug's effect on recurring flare-ups and on symptom severity.

At the meeting, numerous lupus patients testified about their experiences with the disease including constant fatigue, crippling pain and respiratory problems that required lengthy, repeated hospitalization. They expressed frustration with the lack of effective therapies and urged the FDA to approve Benlysta.

Some patients--many of whose travel expenses were reimbursed by the companies--talked about their positive experiences with the drug in clinical trials. Most of the patients who testified were women or teenage girls; lupus largely targets females of child-bearing age.

Because the disease can cause various symptoms, physicians traditionally use different treatments, including anti-inflammatory drugs, steroids, antimalarials and immunosuppressants, all of which have their own potential side effects.

FDA staff scientists also testified and reiterated concerns expressed in an analysis released Friday that associated the drug with an increase in death, and serious side effects including infections and adverse psychiatric events.

Panel member David Blumenthal, assistant professor of medicine at Case Western Reserve University, strongly questioned the presentation of clinical data for Benlysta. The first study, called Bliss-52, only included patients in Asia, Latin America and Eastern Europe, and therefore wasn't representative of lupus patients in the U.S., he said.

The second study, Bliss-76, used patients from the U.S. and Western Europe and showed less effectiveness results than the previous trial after a year. A six-month follow-up showed that some benefits of the drug declined.

Several panel members reiterated Dr. Blumenthal's doubts about the strength of that data. Panel members and the FDA representative expressed concerns that the drug appeared less effective or perhaps even harmful in certain subgroups including African-Americans. Agency scientists also noted that African-American patients' symptoms seemed to worsen on Benlysta. That is particularly troublesome because African-Americans generally have a more aggressive form of lupus, the FDA memo said.

A company-affiliated researcher said at Tuesday's meeting that the results in African-Americans were disappointing and needed to be addressed in future research.

Rheumatologist Murray Urowitz of the University of Toronto told the panel that several factors, including lupus patients' continued use of the steroid prednisone during Bliss tests, make doing any clinical trials on lupus "daunting."

But Dr. Urowitz, who participated in the companies' Benlysta research, added that results from clinical trials indicate good efficacy compared to placebos in relieving some symptoms.

The president of the Lupus Foundation of America, Sandra Raymond, told the advisory committee that patients need new drugs that will improve their quality of life, and recommended Benysta's approval. The foundation, like several lupus groups that urged FDA approval, receives donations from the drug industry.