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Biogen’s new drug for a rare genetic disorder will cost $375,000

Biogen Inc. is based in Cambridge.
Biogen Inc. is based in Cambridge.(Suzanne Kreiter)

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Biogen Inc.’s groundbreaking new drug to treat a rare genetic disorder in children will be priced at about $375,000 a year per patient — its most expensive medicine ever — the Cambridge biotech company said Wednesday.

That will make the treatment, called Spinraza, one of the costliest medicines in the world when it hits the market in January. During its first year of use, the price will be even higher — about $750,000 – because six doses, instead of the subsequent three a year, are initially required, said Biogen spokesman Matt Fearer.

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Analysts project the drug will become a so-called blockbuster treatment, generating annual revenue of at least $1 billion within a few years. Its per patient cost was first reported by Bloomberg News.

Spinraza is the first treatment for spinal muscular atrophy, a devastating condition that affects muscle control in children and is the leading genetic cause of infant death. The disease often limits patients’ ability to breathe, swallow, and move.

As expected, the Food and Drug Administration last week approved the sale of the drug in the US after a trial showed 40 percent of infant patients using the treatment were able to achieve “motor milestones,” such as kicking, rolling, and moving their heads. Forty-three percent of the trial patients who did not receive the drug eventually died, compared with 23 percent of those who were treated with Spinraza.

Biogen said it expects to start shipping the drug to health care providers in about a week. The medicine is delivered through a spinal injection and can be administered at a hospital, pediatrician’s office, or clinical trial site. Michel Vounatsos, Biogen’s incoming chief executive, has said that the launch of Spinraza is the company top immediate priority.

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Fearer said the drug’s high price is consistent with other treatments for rare diseases for which there are no other options. “If there were competing therapies down the road, presumably that would put pricing pressure on it,” he said.

Fearer added that some of the revenue generated by Spinraza will be used “to fund further research to make the next innovation possible.”

Insurers and government agencies are expected to cover the bulk of the drug’s cost for patients. Fearer said Biogen is “adamant about making sure anybody who needs the drug will get it.” He said the company has established a team to help patients limit the amount of their copays and find third-party charitable assistance to cover other costs.

Biogen made a deal with Ionis Pharmaceuticals Inc. in 2012 to work together on Spinraza. The Carlsbad, Calif., company developed the drug in its labs. In August, Biogen paid Ionis $75 million to exercise an option it had to finish work on the drug, and to bring it to market. Ionis can still receive another $225 million, plus royalties, if Spinraza meets certain goals.


Adam Vaccaro can be reached at adam.vaccaro@globe.com. Follow him on Twitter at @adamtvaccaro. Robert Weisman of the Globe staff contributed to this report.