The headline news in autoimmune disease R&D is the launch of the first new treatment for lupus in half a century. Current treatment is limited to steroids and other immunosuppressants, whose side effects can be worse than the symptoms of the disease. Says Decision Resources analyst Michael Latwis: "Lupus has been a very challenging therapeutic area for decades, in part because its symptoms vary so widely that clinical efficacy is exceedingly hard to establish." Yet Human Genome Sciences/GSK's Benlysta (belimumab) was approved by an FDA advisory committee in November—to the cheers of many patients who had testified to their need of the novel (if less than overwhelmingly efficacious) therapy.
A monoclonal antibody delivered by monthly infusions, Benlysta targets an immune protein called B lymphocyte stimulator (BLyS) that causes the hyperactivity of tissue-destroying B cells. BLyS was discovered by Human Genome Sciences in its vast trove of human genes in the glory days of the genomics revolution.
Despite inconsistent results in Phase III trials, the FDA is almost certain to wave Benlysta through to market. "It may only help a small number of patients," says Wolters Kluwer's Ben Weintraub. "Once again, marginal efficacy weighed against major unmet medical needs is likely to carry the day."