Roche reports positive results from latest risdiplam study

(Sharecast News) - Roche announced positive topline results from the pivotal second part of the 'FIREFISH' study on Thursday, evaluating risdiplam in infants aged between one and seven months with type 1 spinal muscular atrophy (SMA).
The Swiss drugmaker siad the primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at 12 months of treatment, assessed by the gross motor scale of the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III).

It said safety for risdiplam in the FIREFISH study was consistent with its known safety profile, with no new safety signals identified.

To date, more than 400 patients had been treated with risdiplam across all studies, with no treatment-related safety findings leading to study withdrawal in any risdiplam trial.

Roche described risdiplam as an investigational survival motor neuron-2 (SMN2) splicing modifier, designed to increase and sustain SMN protein levels throughout the central nervous system and in peripheral tissues.

It was leading the clinical development of risdiplam as part of a collaboration with the SMA Foundation and PTC Therapeutics.

Data from the FIREFISH study would be presented at an upcoming medical congress.

"This large, global trial confirms the efficacy of risdiplam in an advanced and difficult-to-treat population, including many infants whose disease had already progressed significantly before starting treatment," said Roche's chief medical officer and head of global product development, Levi Garraway.

"We are very encouraged by these results and we look forward to sharing them with regulators.

"We also thank the entire SMA community for their continued partnership."

Risdiplam is being studied in a broad clinical trial programme in SMA, with patients ranging from birth to 60 years old, including patients previously treated with SMA-targeting therapies.

Roche said the clinical trial population represented the broad real-world spectrum of people living with the disease, with the aim of ensuring access for all appropriate patients.

On 11 November, the company announced positive results from the second part of 'SUNFISH', which is a study evaluating the efficacy and safety of risdiplam in people aged between two and 25 years with type 2 or 3 SMA.

Also in November, the U.S Food and Drug Administration granted 'priority review' status for risdiplam, with a decision for approval by 24 May.