Genentech (Roche Group) announced positive results of its FIREFISH trial in a recent Business Wire news release. The second part of the study evaluated risdiplam’s efficacy on infants ages one to seven months old, who have been diagnosed with spinal muscular atrophy (SMA) type 1.
The primary end result of the trial was measured by the number of infants who were able to sit without being supported for a minimum of five seconds after being treated with risdiplam for twelve months. The Bayley Scales Third Edition (BSID-III) was used to assess the infants’ developmental function.
The FIREFISH study showed that risdiplam’s safety profile was in line with previous reports. Four hundred patients have now received treatment with risdiplam in Genetech studies. To date, there have been no safety findings related to treatment that caused withdrawal from risdiplam trials.
About Risdiplam
Risdiplam, an investigative therapy for all types of SMA, is designed to increase and sustain liquid survival motor neuron-2 (SMN-2) in the peripheral tissues of the body and in the central nervous system.
Genetech has taken the lead in a wide-ranging program of clinical trials for SMA. Patients in these trials range in age from infants to sixty years of age.
About Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is classified as a rare neurodegenerative disease. The genetic disease negatively impacts motor cells in the spinal cord. Symptoms of SMA are the progressive weakening and wasting of muscles, which limits a patient’s ability to perform basic life tasks, like walking, eating, and even breathing. SMA affects about one in ten thousand babies.
The SMN-1 mutation has been identified as the cause of SMA in most patients. This mutation results in a deficiency of the SMN protein. As additional data is gathered, it appears more likely that SMA may be a multi-system disease. The assumption is that the body’s loss of protein will have an effect on its functioning. Additional information about SMA is available here.
About FIREFISH (NCT02913482)
FIREFISH Part 1 assesses risdiplam relative to optimum dosage. FIREFISH Part 2 focused on assessing the drug’s efficacy. This part of the study reviews the number of infants who were able to sit without support after being treated with the drug for twelve months.
Three other Genentech trials for risdiplam are:
- JEWELFISH (NCT03032172)
- RAINBOWFISH (NCT03779334)
- SUNFISH (NCT02908685)
Neuroscience and Genentech
Neuroscience applies to all sciences that deal with the structure or function of the nervous system and the brain. It is the primary focus of Genentech and Roche. Genentech is a major biotechnology company that was founded over forty years ago.
