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First lupus drug in 52 years nears approval
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The FDA Arthritis Advisory Committee voted 13-2 with no abstentions in support of a biologic license application for 10 mg/kg IV belimumab with the proposed indication of reducing the disease activity of active auto-antibody-positive systemic lupus erythematosus in adults.
The application for the intravenous drug, 125370, was co-sponsored by Human Genome Sciences Inc. (HGSI), Rockville, Md., and Glaxo SmithKline (GSK), London.
If approved, the investigational human monoclonal antibody drug belimumab (proposed to be marketed as Benlysta) will become the first in a new class of drugs called BLyS-specific inhibitors to be marketed in the United States.
“I think it’s very exciting that this is the first [lupus] drug to be approved after 5 decades, but I do have some reservations in that I don’t think this is a magic bullet,” said Maria E. Suarez-Almazor, MD, professor and section chief of rheumatology at Houston’s MD Anderson Cancer Center, who voted in favor of approving belimumab.
Placebo-controlled trials
The novel lupus drug was studied in three placebo-controlled trials, including two trials spanning 52 and 76 weeks. In a 10-5 vote that was separate from the approval vote, the committee accepted results of efficacy studies in which belimumab IV 1 mg/kg and 10 mg/kg were compared with placebo with a primary endpoint of statistically significant improvement at 52 weeks in the systemic lupus erythematosus (SLE) responder index (SRI).
The SRI is a composite score that also accounts for no worsening of clinical symptoms. The 10 mg/kg dose met the primary endpoint in both phase 3 trials, according to the HGSI/GSK briefing document.
The FDA granted belimumab priority review designation and assigned it a Prescription Drug User Fee Act target date of December 9, 2010, according to a HGSI/GSK press release.
Efficacy
No drugs for treating SLE in adults have been introduced to the market in the last 52 years, and none have ever specifically been approved for SLE. Aspirin, corticosteroid and hydroxychloroquine are approved for treating lupus, as mentioned during the meeting.
Panel members agreed in a 14-1 vote that the product’s safety profile was sufficient as it pertained to the target population who were already on standard therapies for the condition. Across all three studies, 14 deaths occurred and nearly all patients experienced one adverse event, with infection being the most commonly reported event.
However, physicians, researchers and others on the panel were less convinced of belimumab’s efficacy. Should the FDA approve the medication, most panelists called for clearer labeling than what HGSI/GSK have proposed. Many requested language on the labeling that would reflect that the drug’s interactions with other therapies was not studied and that patients with central nervous system and renal conditions were excluded from the pivotal trials, hence their response to belimumab could not be determined from the study results.
One stumbling block concerning efficacy, was how findings from the studies the sponsor conducted in European and Asian populations might apply to North American lupus patients for which U.S. rheumatologists would typically be prescribing the new therapy.
The panelists also heard testimonies from more than 20 lupus patients, their families, and others.
“I found the personal comments and stories we heard today particularly compelling, and I see this as an important opportunity to advance a field of medicine and the care of a previously underserved and understudied and poorly treated population,” said Robert Kerns, PhD, professor of psychiatry, neurology and psychology at Yale University, who voted in favor of approving belimumab.
The rheumatologists have led using existing medications to manage lupus patients. They have been successful at extending life, but at a cost that’s unacceptable to this nation. There is a huge lupus population that falls into the category of having non-life-threatening lupus. They use the same medications as those with life-threatening forms and suffer the same indignities. Today it’s past time to turn the corner. We must have earlier diagnosis and treatments to bring lupus under control. It is time to usher in a new era of enlightenment in lupus research.
— Sandra C. Raymond
President and CEO of the Lupus Foundation of America
Washington, D.C.
Delivered during the open public hearing portion of the FDA meeting
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