Genentech Gains Breakthrough Status for Scleroderma Treatment

The FDA has granted breakthrough therapy status for Genentech's scleroderma treatment Actemra, guaranteeing the drug a six-month review rather than the usual 10 months.

The designation of Actemra (tocilizumab) underscores the need for treatment for the degenerative disorder, which afflicts 75,000 to 100,000 people in the U.S., the majority of them women, says Sandra Horning, chief medical officer and head of medical development at the South San Francisco, Calif., company.

Breakthrough therapy status is granted for drugs that are intended to treat serious or life-threatening diseases and that have shown substantial improvement over existing therapies in early-stage clinical trials.

The FDA based its decision on data from a Phase 2 study that showed continued slowing of skin thickening between weeks 24 and 48.

There are currently no FDA-approved treatments for scleroderma, which, according to Genentech, has the highest mortality of any autoimmune rheumatic disease. Its key symptoms are skin thickening and hardening.

The drugmaker has launched a double-blind placebo-controlled Phase 3 trial based on the Phase 2 results and the unmet need in patients.

Actemra, a humanized interleukin-6 receptor antagonist, is already approved to treat adult patients with moderately to severely active rheumatoid arthritis who have used one or more drugs without relief. It is also able to treat juvenile arthritis. — Charlotte Astor