Spinraza Wins 'Best Case Approval'; Leerink Raises Targets On Biogen, Ionis

The FDA has approved Spinraza for the treatment of all types of spinal muscular atrophy. The approval comes six months earlier than expected and with a label that is “as broad as even the most optimistic patient or investor could have hoped for,” Leerink’s Geoffrey C. Porges said in a report.

Price Targets Raised

Porges maintains an Outperform rating on Biogen Inc BIIB, while raising the price target from $355 to $368.

In a separate note, Leerink’s Paul Matteis reiterated a Market Perform rating on Ionis Pharmaceuticals Inc IONS, while raising the price target from $40 to $47.

Improved Prospects

Given the broad SMA label, many children and adolescents suffering from the more severe and progressive forms of the disease would likely pursue treatment in 2017 and 2018, Porges mentioned. He added that payers also have limited grounds for denying access, given the significant benefit shown to date.

Biogen planned to make Spinraza available for shipment within one week, which means that the company would be able to record revenue for most of 2017, as compared to the earlier expectation of half the year or less.

Given the approval, the probability of success has been raised to 100 percent for all SMA, Matteis noted. He added that although the FDA had asked for some warnings to be included, the safety language in the label is unlikely to have any commercial impact, “given the substantial need for treatment in SMA, the debilitating nature of the disease, the strength of the nusinersen efficacy data, and the direct intrathecal delivery + much lower patient exposure of Spinraza relative to other antisense medicines.”