Risdiplam approval looks certain after more positive data

23 January 2020

Swiss pharma giant Roche (ROG: SIX) has announced positive top-line results from the pivotal Part 2 of the FIREFISH study, which is testing risdiplam in young infants with Type 1 spinal muscular atrophy (SMA).

The primary endpoint was the proportion of infants sitting without support for at least five seconds after one year, following a commonly-used scale.

Risdiplam, an orally administered liquid, is an investigational survival motor neuron-2 (SMN-2) splicing modifier. Roche leads the clinical development of the option as part of a collaboration with the SMA Foundation and PTC Therapeutics (Nasdaq: PTCT).

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