With 'remarkable' data, Biogen's Spinraza sharpens its case against Novartis gene therapy

Biogen credited spinal muscular atrophy (SMA) drug Spinraza as its main growth driver last year, and the company aims to keep it that way—even in the face of new competition from Novartis gene therapy Zolgensma.

So Biogen is racking up data to bolster Spinraza's case, and at the Cure SMA conference on Monday, it rolled out "remarkable" numbers in patients treated before symptoms develop. And those data show just how the drug has established itself as the "foundation of treatment" in the disease, a Biogen executive said.

In an ongoing study of 25 SMA patients on Spinraza called Nurture, all are alive and don’t require permanent breathing assistance from a ventilator. Investigators followed the patients, who were mostly likely to develop Type 1 or 2 SMA, for up to nearly four years.

All patients received their first treatments before six weeks of age, and the "overwhelming majority" are achieving motor milestones at ages typical for babies without SMA, the drugmaker said.

For example, all patients in the Nurture study are able to sit independently, Biogen reports. Type 1 SMA patients wouldn't be able to accomplish that feat in the natural history of the disease. And 88% of patients are walking independently; without treatment, Type 1 or 2 patients wouldn’t ever be able to walk on their own. 

"This is a remarkable result, well beyond anything that has been previously reported or ever seen in the natural history of patients with SMA," Biogen’s Wildon Farwell, executive director and head of late stage neuromuscular, told FiercePharma.

Without treatment, patients typically don't live beyond the age of 2 or require permanent ventilation before then, the company said.

The data presentation comes early in Novartis' competing launch for gene therapy Zolgensma. Novartis scored an FDA approval in May for patients under 2, so Biogen is working to defend Spinraza in young patients.

And Biogen seems to have a tagline for its defense. Biogen launched Spinraza in early 2016, and in the years since, the drug has become the "foundation of treatment," Farwell said. The drugmaker itself called Spinraza a "foundation of care" in its Monday press release.

"Spinraza now is being used by more than 7,500 patients around the world," Farwell said, adding, "As that has occurred, awareness and ability to diagnose these patients has improved as well."

Biogen caught early criticism over the drug’s high price of $750,000 for the first year and $375,000 for subsequent years, but sales have far outpaced early expectations. In 2018, the drug's global sales grew to $1.7 billion. Biogen cited the launch as its primary growth driver in 2018.

Zolgensma, a one-time SMA treatment, carries the world’s highest pharma price tag at $2.125 million. Despite the sticker, cost watchdogs have signed off, citing the drug's efficacy data in presymptomatic patients.