Atlanta-based biopharmaceutical company Tikvah Therapeutics announced this week that it has received orphan drug status from the U.S. Food and Drug Administration for use of sodium phenylbutyrate as a treatment for spinal muscular atrophy (SMA).  The orphan drug status gives Tikvah seven years of market exclusivity for use of phenylbutyrate for SMA treatment.

Findings from in vitro studies by Tikvah, as well as pilot clinical work, suggest that phenylbutyrate treatment in SMA patients may improve motor function.

Tikvah will be working in conjunction with the FDA, and collaborative clinical trial groups focused on SMA to develop well-controlled multicenter trials to fully evaluate sodium phenylbutyrate in the treatment of SMA. Tikvah Therapeutics is also supporting research on new diagnostic approaches and new therapeutic approaches to the treatment of SMA and allied neurodegenerative diseases such as ALS (amyotropic lateral sclerois) and MS (multiple sclerosis).

Click here to view Tikvah’s entire announcement.