Genentech’s SMA Type 1 Data Strengthen Case Backing Risdiplam Approval

With top-line, pivotal data showing efficacy in infants with type 1 spinal muscular atrophy (SMA), Genentech Inc. has bolstered the case for approval of risdiplam, a survival motor neuron-2 (SMN-2) splicing modifier under review at the US Food and Drug Administration with a 24 May action date.

On 22 January, the Roche affiliate reported that risdiplam demonstrated statistical significance in infant patients with type 1 SMA aged one to seven months for the ability to sit up without support for at least five seconds, which the company called a medically meaningful motor milestone improvement. The top-line data were from part two of the pivotal FIREFISH study; part one of that study investigating risdiplam’s safety and optimal dosing were included in the new drug application at the FDA, along with safety and efficacy data from the SUNFISH study in patients with type 2 or type 3 SMA