Isis Pharmaceuticals Announces Phase 3 Study for Infants with SMA

Isis Pharmaceuticals is planning to start a "pivotal" phase 3 study of its ISIS-SMN Rx drug on infants with spinal muscular atrophy (SMA).

In an Aug. 1 news announcement, Isis Pharmaceuticals said it was planning to start giving the drug to the first infant in the study "within the next few weeks." Another study involving children with SMA is scheduled for later in the year.

The ISIS-SMN Rx drug "is designed to alter the splicing of a closely related gene - SMN2 - to increase production of fully functional SMN protein," the company said in its news release. It added that the U.S. Food & Drug Administration had granted "orphan drug status" - given to potential treatments for rare diseases - and fast tracked the drug.

Ultimately, the goal of the drug would be "survival or permanent ventilation" of the children involved. Spinal muscular atrophy is a motor neuron disease that leads to wasting away of muscles throughout the body. In its most severe forms, it's diagnosed in infancy and can lead to death within a few years or less.

"Families of SMA (FSMA) is pleased that Isis is advancing to the next phase of clinical trials for ISIS-SMN Rx," said FSMA President Kenneth Hobby in the news announcement. "Controlled trials are the gold standard in proving the safety and efficacy of any drug. SMA is a devastating disease with no current therapeutic options. Families of SMA applauds Isis for progressing its developmental program in an expedient manner, and looks forward to additional trials in patients with SMA beginning later in 2014."

August is SMA awareness month.

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Laurie Watanabe is the editor of Mobility Management. She can be reached at