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Genentech : Risdiplam Shows Improvement In Survival In Infants With Type 1 SMA

Genentech, a member of the Roche Group (RHHBY), said that its risdiplam showed significant improvement in survival and motor milestones in infants with type 1 spinal muscular atrophy.

The company presented 1-year data from FIREFISH Part 2 study that evaluated risdiplam in infants aged 1-7 months old with symptomatic Type 1 spinal muscular atrophy.

The company noted that the study met its primary endpoint by demonstrating a significant increase in motor milestones in infants aged 1-7 months after 12 months of treatment.

In November 2019, the U.S. Food and Drug Administration granted Priority Review for risdiplam. A decision on approval is expected by August 24, 2020.

Spinal muscular atrophy or 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.

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 (SMN-1) 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.

In a separate press release, Genentech announced new analyses of Phase III OPERA I and OPERA II studies, as well as the open-label extensions, showing that Ocrevus or ocrelizumab treatment reduced the risk of disease and disability progression in Relapsing Multiple Sclerosis or RMS and primary progressive MS or PPMS.

The new analyses add additional evidence to the benefit-risk profile of Ocrevus, including the impact of MS on people's daily lives.

The Post-hoc analysis from 6 years of Phase III open-label extension studies showed Ocrevus treatment reduced the risk of needing a walking aid by 49% in relapsing multiple sclerosis (RMS) patients compared with patients who switched from interferon beta-1a two years later.

A separate analysis showed Ocrevus slowed thalamic volume loss in patients with RMS and primary progressive MS (PPMS) vs. interferon beta-1a and placebo, respectively.

Multiple sclerosis or MS occurs when the immune system abnormally attacks the insulation and support around nerve cells in the brain, spinal cord and optic nerves, causing inflammation and consequent damage. This damage can cause a wide range of symptoms, including muscle weakness, fatigue and difficulty seeing, and may eventually lead to disability.

Relapsing-remitting MS (RRMS) is the most common form of the disease and is characterized by episodes of new or worsening signs or symptoms followed by periods of recovery.

Approximately 85 percent of people with MS are initially diagnosed with RRMS. The majority of people who are diagnosed with RRMS will eventually transition to secondary progressive MS (SPMS), in which they experience steadily worsening disability over time.

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