Biogen's Spinraza scores later-onset SMA data for stepped-up push with payers

Biogen reported new phase 3 data for its superpricey Spinraza on Monday.

Hoping to win over careful payers, Biogen has new data to support its superpricey spinal muscular atrophy (SMA) drug Spinraza.

In new phase 3 data from a study in children with later-onset SMA, patients on Spinraza “demonstrated a highly statistically significant and clinically meaningful improvement in motor function” over their counterparts who received a sham treatment, the Cambridge, Massachusetts-based biotech said in a Monday release.

Biogen won approval for the med back in December and sparked criticism by pricing the groundbreaking injection at $750,000 for the first year, a figure that falls to $375,000 for subsequent years. In an earlier study of infantile-onset (type 1) patients, a higher percentage of children on Spinraza survived compared with untreated patients.

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Data from the newly finished study, Cherish, weren’t available at the time, but the FDA nonetheless approved the med for treatment of all SMA types.

So far, Anthem, UnitedHealth, Humana, Aetna and Blue Cross Blue Shield have agreed to cover the med for type 1 SMA patients, but the picture gets more complicated for other types, according to a presentation last month from Evercore ISI analyst Umer Raffat. Several other insurers have yet to release their Spinraza guidelines.

Speaking about the new data with the Boston Globe, Biogen Chief Medical Officer Alfred Sandrock Jr., M.D., Ph.D., said the company hopes insurers will “update their policies” to include coverage for older children.

In the Cherish study, investigators documented a 4.9-point difference—as measured on the expanded Hammersmith Functional Motor Scale—between patients on Spinraza and those who didn’t receive treatment. The scale evaluates motor function in SMA patients, with scores ranging between 0 and 66, according to Biogen’s website Together in SMA.

Shortly after the approval, Biogen’s pricing came under fire on Twitter, where critics reached out to high-profile political figures, including then President-elect Donald Trump, asking for action. Biogen stood by its decision; a spokesperson said the company “carefully considered” Spinraza’s sticker price.

RELATED: Biogen’s Spinraza sparks pricing concerns as Trump renews attack on pharma

More recently, after conducting a survey of 50 neurologists who treat 675 patients, Instinet analyst Christopher Marai wrote last week that the launch “may be progressing better than consensus” estimates. He predicted $29 million in first-quarter Spinraza sales due to “broad utilization” early in the launch.

More than 100 patients have been treated with Spinraza, according to Marai. Among those treated, 40% are type 1 SMA patients, while 40% are type 2 and 20% are type 3, the survey found.

In first-quarter results unveiled early Tuesday, Biogen reported $47 million in Spinraza sales for the period, handily beating consensus estimates of $16 million. As of April 21, 100 commercial insurance plans and 65 Medicaid plans had approved individual uses of the med, according to the company's first-quarter earnings presentation.

Speaking with analysts on Tuesday's call, Biogen execs said 75% of commercially covered lives in the U.S. are on an insurance plan with access to Spinraza, with half of those on a plan with broad coverage. The company is “presenting data tonight that will hopefully start to get more broad coverage,” one exec said, referring to the Cherish study.

In Europe, the company is working on "prelaunch" efforts following a European Medicines Agency committee's positive recommendation on Friday. Biogen is "prioritizing" countries such as Germany and the U.K., where it believes it'll be able to secure reimbursement before other markets, execs said Tuesday.

Elsewhere around the world, Biogen is expecting approvals in Canada and Japan this year, and has filed for Spinraza approval in Australia. It's anticipating filing for approval in 10 other countries this year.

Notably, to ensure patient access, the company gave away 25% of Spinraza injections for free in the first quarter.

Editor's note: This story was updated to include information on Spinraza's launch from Biogen's first-quarter results.