- Major U.S. insurer Anthem has restricted coverage of Biogen's newly approved spinal muscular atrophy drug to only those patients with the most severe form of the genetic disease, raising questions of whether the medicine's sky-high price tag will slow sales.
- Anthem judged Biogen's drug, called Spinraza (nusinersen), medically necessary for patients with type 1 spinal muscular atrophy (SMA), despite a broad label covering all adult and pediatric patients.
- Furthermore, the insurer will require evidence of clinically meaningful improvements for continuation of treatment beyond 6 months post-initiation and for every subsequent 6-month period.
Spinraza is the first drug approved by the Food and Drug Administration to treat SMA, a fact which has fueled expectations for a strong launch.
Anthem's decision to limit coverage could dampen those expectations somewhat though, particularly if other insurers follow suit. Biogen does plan to report new clinical evidence showing Spinraza's efficacy in type 2 SMA soon, which could give insurers reason to cover the drug more broadly.
Patients with SMA suffer from progressive muscular atrophy and weakness resulting from the loss of motor neurons in the spinal cord and lower brain stem. The most severe form of SMA — type 1 — represents about 60% of SMA diagnoses, according to Anthem, and can result in paralysis and difficulty breathing or swallowing. Most infants with type 1 die by their second year.
For coverage, Anthem will require documentation of confirmatory diagnosis by genetic testing and evidence of SMA-associated symptoms prior to 6 months of age. All other uses, including for a related SMA disease labeled non-5q, will be considered "investigational and not medically necessary."
Jefferies, an investment firm, currently forecasts revenues of $264 million for Biogen from sales of Spinraza in 2017. (Biogen licensed the drug from Ionis Pharmaceuticals.) Removing type 2 and type 3 SMA patients from models completely could significantly lower those figures, Jefferies said, although that would be a worst-case scenario.
Biogen had caused a stir when it set the price of Spinraza at $125,000 per vial, which translates to a $750,000 price tag in the first year of treatment and $375,000 for each following year.
It is not clear yet if other insurers will follow Anthem's lead.
A spokesperson for Humana said the company was "still evaluating the clinical merits" of Spinraza. Requests for comment from Aetna and Cigna were not returned by time of publication.