Biogen courts controversy with $750k-a-year rare disease drug

First, the good news: the FDA has approved Spinraza, the first drug to treat children and adults with the rare and often fatal muscle wasting disease, spinal muscular atrophy (SMA).

But now the bad news for patients and healthcare systems:  Biogen's list-price annual cost of Spinraza (nusinersen) will be $750,000 for six jabs in the first year of treatment.

Although Biogen has a stronger case than some to demand a high premium for its drug, Spinraza is likely to keep the burning issue of US drug prices alight in 2017.

Biogen will offer the spinally injected drug at half price after the first year, as from then on only three doses are required annually. But the high cost is likely to face pushback from US insurers and pharmacy benefit managers.

SMA is a genetic disorder caused by a loss of motor neurons in the spinal chord and brainstem, leading to weakness and wasting of muscles used for crawling, walking, sitting  up, and head movement. In severe cases, breathing and swallowing are affected.

Looking for new revenues as its big-selling multiple sclerosis drug Tecfidera (dimethyl fumarate) faces an increasingly tough market, Biogen will likely try and push for similar high prices in Europe, where regulators are still reviewing Spinraza.

Biogen, its development partner Ionis,  and the FDA received plaudits on Twitter for the achievement – with many praising the pharma companies for the strong clinical data they had produced. The regulator received glowing reviews for its fast approval decision.

The FDA pushed Spinraza through its review process in about a month and half, on the basis of phase 3 data showing patients who received the drug experienced a highly statistically significant improvement in motor function, compared with those who did not receive it.

One expert contrasted Biogen’s filing with the flimsy data the FDA controversially accepted last year to approve Sarepta’s Exondys 51 (eteplirsen) for Duchenne muscular dystrophy, another rare inherited muscle wasting disease.

https://twitter.com/EricTopol/status/812481704308850688

But whether patients will be able to access Biogen’s drug, available this week, is a completely different matter. At a time when president-elect Donald Trump has said that he wishes to tackle the issue of high drug prices, payers are becoming increasingly sensitive about the issue.

One analyst, Leerink's Geoffrey Porges, said the Biogen's price for Spinraza could become as controversial as Gilead's Sovaldi (sofosbuvir) and similar drugs - which cure hepatitis C but cost tens of thousands of dollars for a 12-week course.

There are concerns that the high price for Spinraza will lead to rationing, although a Biogen spokesperson said it recognises "that treatment cost and access are critical concerns for patients, providers, payers and policy makers."

"We are working to help ensure that no patient will forego treatment because of financial limitations or insurance status," the spokesperson added in an emailed statement.