Top 10 for '09: No. 9

No. 9: Belimumab Buzz

There were few notable FDA approvals for "new molecular entities" (NMEs) in 2009:

• Urolic (febuxostat; Takeda) is the first new treatment for gout in more than 40 years. (But come on: it's gout.)
• Simponi (galimumab; Centacor/Ortho Biotech) is yet another TNF inhibitor for rheumatoid arthritis.
• Effient (prasugrel; Lilly) is an antiplatelet therapy poised to take market share away from chemically related Plavix (clodigrel; BMS/sanofi-aventis)—but only in the setting of angioplasty and stent placement.
• Sabril (vigabratin; Lundbeck) is the first anticonvulsant approved for the treatment of infantile spasms, a rare and devastating form of pediatric epilepsy.

And there were drugs approved for even rarer disorders: Ilaris (canakinumab; Novartis), for the cryptic cryopyrin-associated periodic syndromes; and Kalbitor (ecallantide; Dyax) for hereditary angioedema. Add to those: approvals for yet another statin, another fluoroquinolone, another antidiabetic agent...and you get the idea of the ho-hum-ness of 2009.

In pharma, expectations currently run low for groundbreaking treatments, whether for rare or common disorders. One exception, however, may be Benlysta (belimumab). The human monoclonal antibody, codeveloped by Human Genome Sciences and GSK, inhibits the activity of B-lymphocyte stimulator (BLys), which otherwise promotes the survival of antibody-producing B cells. The drug's mechanism of action supports its use in autoimmune disorders, like systemic lupus erythematosus (SLE).

To that end, favorable data from 2 phase 3 trials of Benlysta (BLISS-52 and BLISS-76) in patients with SLE were reported in July and November. The positive clinical data are notable, because there hasn't been a new treatment for the 1.5 million Americans with lupus in about 30 years, says the Lupus Foundation of America. Even the almighty Rituxan (rituximab; Genentech/Roche) bit the dust this year in phase 3 studies of patients with SLE. And another great hope for SLE, CellCept (mycophenolate mofetil; Roche), died a death of noninferiority after it performed no better than cyclophosphamide for the induction treatment of lupus nephritis.

Excitement over Benlysta should be tempered, however, by the fact that neither phase 3 study has been published in a peer-reviewed journal—yet. Human Genome Sciences indicates that applications for the approval of Benlysta in the United States and Europe will be submitted during the first half of 2010. 

TNF = tumor necrosis factor.