Awaiting FDA nod, Novartis touts latest data on gene therapy Zolgensma

As Novartis waits for FDA action on its gene therapy Zolgensma, expected this month, the company has rolled out yet another round of data on the spinal muscular atrophy treatment.

Novartis’ AveXis, which developed the gene therapy, touted interim data through March 8 from a phase 1 study in Type 2 SMA patients, plus later-stage studies in Type 1 and presymptompatic SMA patients. In the Strong study, which is testing Zolgensma in patients with Type 2 SMA, the drug demonstrated “rapid motor function gains and milestone achievements,” AveXis said. 

All 30 patients in the Strong study were still alive, the company reported. Investigators tracked two treatment-related adverse events, both transaminase elevation. But even with just 6.5 months of follow-up after treatment, 10 patients achieved 22 motor milestones. 

“With an average of just over six months of data available for these Type 2 patients following treatment with Zolgensma, we are pleased to see they are achieving motor milestones, including the ability to stand and walk,” AveXis’ chief medical officer Olga Santiago said in a statement.  

In the Str1ve study in Type 1 patients, Zolgensma “continued to show prolonged event-free survival, increases in motor function and significant milestone achievement.” Lastly, the drug helped presymptomatic patients with motor milestone achievements in the phase 3 Spr1nt study, AveXis said. 

Type 1 SMA is the most common form of the disease and occurs in the first six months of life. Sixty-eight percent of children with the disease don’t live to their second birthday, according to SMA News Today. Type 2 SMA occurs between 7 months and 18 months of age. Those patients may be able to sit independently, but most can’t stand or walk without assistance. Novartis is seeking approval in Type 1 SMA, but it's testing the drug in various patient groups. 

The data release came over the weekend at the American Academy of Neurology annual meeting in Philidelphia. Novartis submitted its drug—purchased in the AveXis buyout—to the FDA last December to address the genetic cause of SMA Type 1. The FDA tagged the drug with a priority review, so it’s expected to act on the med this month. 

Altogether, the data “represent a growing body of evidence that support the use of Zolgensma as a potential foundational therapy for the treatment of SMA across a variety of populations,” AveXis president David Lennon said in a statement. 

Cantor Fitzgerald analysts wrote that the Type 2 data are still early and “hard to draw competitive efficacy conclusions" against the competition in the field, including Biogen's Spinraza. They’re “cautious that these data are mature enough to support a broad label along with Type 1 approval expected this month.” Still, the analysts wrote that Zolgensma’s Type 1 data are “compelling.”  They're also keeping an eye out for data on risdiplam in all SMA types from Roche and PTC Therapeutics, expected Tuesday.

For their part, SVB Leerink analysts wrote that they “believe the favorable treatment profile emerging for [Zolgensma] across Type 1 SMA, later-onset SMA, and presymptomatic SMA will resonate with both physicians and investors alike, and these data will be supportive for registration across all of these patient groups.” 

As it awaits an FDA decision, Novartis has been working to build its case for the one-time treatment expected to carry a price tag in the millions of dollars. Last month, the company touted interim data from the Str1ve study showing all but one of the 22 Type 1 SMA patients treated with the drug were alive and had not suffered serious complications as of the end of September.  

The drugmaker has said its drug could be cost-effective with a price tag of $4 million to $5 million, but analysts expect the price to be around $2 million.