FDA grants priority review to Roche?s risdiplam for spinal muscular atrophy
[caption id="attachment_9277" align="aligncenter" width="747"] Press Release[/caption]
- Filing submission includes 12-month data from pivotal FIREFISH and SUNFISH trials in a broad population of people living with Types 1, 2 or 3 SMA
About spinal muscular atrophy
Spinal muscular atrophy (SMA) is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. It is the most common genetic cause of infant mortality and one of the most common rare diseases, affecting approximately one in 11,000 babies. SMA leads to the progressive loss of nerve cells in the spinal cord that control muscle movement. Depending on the type of SMA, an individual?s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost. SMA is caused by a mutation in the survival motor neuron 1 (SMN1) gene that results in a deficiency of SMN protein. SMN protein is found throughout the body and increasing evidence suggests SMA is a multi-system disorder and the loss of SMN protein may affect many tissues and cells, which can stop the body from functioning.About risdiplam
Risdiplam is an investigational, orally administered liquid survival motor neuron-2 (SMN-2) splicing modifier for SMA. It is designed to increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body. It is being evaluated for its potential ability to help the SMN2 gene produce more functional SMN protein throughout the body. Risdiplam is currently being evaluated in four multicenter trials in people with SMA.- FIREFISH (NCT02913482) ? an ongoing open-label, two-part pivotal clinical trial in infants with Type 1 SMA.
- SUNFISH (NCT02908685) ? a two-part, double-blind, placebo-controlled pivotal clinical trial in children and young adults (two to 25 years old) with Type 2 or 3 SMA.
- JEWELFISH (NCT03032172) ? an open-label exploratory trial in people with SMA Type 1, 2 or 3, aged 6 months to 60 years who have been previously treated with SMN-targeting therapy, gene therapy or olesoxime. The study is currently recruiting.
- RAINBOWFISH (NCT03779334) ? an open-label, single-arm, multicentre study, investigating the efficacy, safety, pharmacokinetics and pharmacodynamics of risdiplam in babies (~n=25), from birth to six weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study is currently recruiting.
About Roche in neuroscience
Neuroscience is a major focus of research and development at Roche. The company?s goal is to develop treatment options based on the biology of the nervous system to help improve the lives of people with chronic and potentially devastating diseases. Roche has more than a dozen investigational medicines in clinical development for diseases that include multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer?s disease, Huntington?s disease, Parkinson?s disease and autism.About Roche
Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people?s lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare ? a strategy that aims to fit the right treatment to each patient in the best way possible. Roche is the world?s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the eleventh consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI). The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2018 employed about 94,000 people worldwide. In 2018, Roche invested CHF 11 billion in R&D and posted sales of CHF 56.8 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit?www.roche.com. *MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMAAll trademarks used or mentioned in this release are protected by law.