FDA Green-Lights HGS and GSK’s Systemic Lupus Erythematosus Drug Benlysta

Companies hope to make BLyS inhibitor available to physicians within two weeks.

FDA has approved Human Genome Sciences (HGS) and GlaxoSmithKline’s (GSK) Benlysta® (belimumab) for the treatment of adult patients with active, autoantibody-positive systemic lupus erythematosus (SLE) who are receiving standard therapy. Regulatory review in the EU is in progress, and a decision by the EMA is expected during the second half of 2011. Positive data from two pivotal Phase III studies, Bliss-52 and Bliss-76, were reported back in 2009 and demonstrated that Benlysta therapy reduced SLE activity. The firms hope to make the drug available to physicians in the U.S. within a couple of weeks.

FDA approval represents “a moment of enormous relief for people with lupus and a landmark achievement,” comments Margaret G. Dowd, president of the Lupus Research Institute. “Benlysta’s winding path to FDA approval is a triumph for the dedicated pursuit of innovative science and smart trial design in achieving solid results in the complex autoimmune disease of lupus.”

U.S. clearance does come with limitations of use. Benlysta’s efficacy has not been evaluated in patients with severe active lupus nephritis or severe active central nervous sytem lupus, or in combination with other biologics or intravenous cyclophosphamide, and so it is not recommended for these patients.  

HGS estimates that some five million people worldwide, including approximately 1.5 million in the U.S., suffer from various forms of lupus including SLE. Benlysta is designed to block the activity of B-lymphocyte stimulator (BLyS), a protein discovered by HGS that the firm claims is needed for the survival and development of B lymphocytes into mature plasma B cells. Benlysta effectively binds to BLyS, so inhibiting the stimulation of B cell development and restoring the potential for autoantibody-producing B cells to undergo the normal process of apoptosis.

HGS and GSK have been co-developing Benlysta under an agreement signed in 2006. HGS conducted the Benlysta Phase III trials with assistance from GSK. The companies will share equally in Phase III/IV development costs, sales, and marketing expenses, as well as profits from sales.

The partners are meanwhile evaluating a range of potential additional indications for Benlysta, including vasculitis and post-renal transplant. Small proof-of-concept studies are either ongoing or are planned for diseases including Sjögren syndrome, Waldenstrom disease, and pretransplant and idiopathic thrombocytopenic purpura, HGS states.

In July 2010 HGS and Lonza inked a manufacturing deal through which the latter will produce Benlysta on a commercial scale. At the time HGS said that although its own manufacturing facility had the capacity to supply the drug worldwide for the first two or three years following launch, it believed worldwide demand would eventually become too high for production in house.