A new gene therapy on the horizon for SMA

A new intravenous (IV) medicine, Zolgensma (onasemnogene abeparvovec-xxxx), is getting a priority review from the United States' Food and Drug Administration (FDA) for the treatment of spinal muscular atrophy (SMA) type 1, in infants up to 9 months old.

SMA is a rare genetic disease which decreases muscle function as it progresses. Type 1 of this disease is the most frequent and severe form. Infants with SMA type 1 usually show symptoms shortly after birth, and, if left untreated, they may not be able to lift their heads, sit up without help, or breathe, cough or swallow normally. The only medicine currently approved for SMA involves a lifelong and invasive treatment consisting of periodic injections into the spinal fluid.

On the other hand, the new medicine is a gene replacement therapy which is intended as a “one-time, potentially curative therapy” according to David Lennon, president of the company that developed Zolgensma (onasemnogene abeparvovec-xxxx) (AveXis). Dr Lennon also stated that their new medicine “addresses the genetic root cause of SMA without the need for repeat dosing” and therefore "represents a potentially significant therapeutic advance for these patients and their families”. Zolgensma (onasemnogene abeparvovec-xxxx) could become the second therapy for SMA should it win approval; Spinraza, the first, was approved by the FDA for pediatric and adult patients in December 2016.

For more details, please read the full article on SMA News Today.