PTC Therapeutics reports results of risdiplam in spinal muscular atrophy

PTC Therapeutics announced interim clinical data from the Part 1, open-label studies of FIREFISH and SUNFISH demonstrating the ‘benefit’ of risdiplam for the treatment of Type 1, 2 and 3 spinal muscular atrophy. The results showed that patients across all SMA types benefited from an oral systemic therapy indicated by increases in developmental motor milestones. Risdiplam was well tolerated at all doses across studies to date. The SMA program is a collaboration between PTC, the SMA Foundation, and Roche. In Part 1 of the FIREFISH study, at Day 245 of treatment, 43% or 6/14, of infants were able to sit with or without support, including three who achieved unassisted sitting. Natural history indicates that Type 1 SMA babies never achieve this milestone. In Part 1 of the SUNFISH study in Type 2 and 3 SMA patients, 63% or 19/30, of patients treated with risdiplam for at least one year achieved a median increase in motor function of 3.13 points versus baseline. Typically patients with Type 2 or 3 SMA decline by 0.85 to 0.67 point per year. The pivotal portion of the SUNFISH clinical study has completed enrollment. Patients enrolled in the SUNFISH trial have a broad age range (2-24 years; median age 8 years) and with broad functional characteristics.

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