Another blow to Zolgensma, but good news for Biogen and Roche?

If last week’s forecast-busting Zolgensma sales number was bad news for Biogen, then a clinical hold on an intrathecal version of the Novartis gene therapy could be something of a fillip. It is too soon to know the full implications of the FDA’s actions, triggered by a preclinical safety signal, but a delay to approval of Zolgensma’s new formulation is surely the best case scenario. An intravenously administered version of the spinal muscular atrophy product is currently approved to treat babies up to two years old; the intrathecal product was designed to broaden usage to older patients. Approval here would have put Zolgensma on a similar footing to Biogen’s SMA therapy, Spinraza, which is indicated for all age groups. Forecasts show that the sellside expects sales of Spinraza to peak next year, as competition from Zolgensma and Roche’s risdiplam chip away at Biogen’s dominance. The Novartis setback could also be good news for risdiplam’s launch: Roche is due to file for approval in all patients this year. For Novartis, however, this could turn into yet another damaging blow to its high-profile gene therapy, in the wake of a data manipulation scandal and delays to approval in Japan and Europe.