Roche presents new data at World Muscle Society (WMS) 2021 highlighting new advances for people living with rare neuromuscular disorders

New data show pre-symptomatic babies with spinal muscular atrophy (SMA) treated with Evrysdi maintained the power to swallow
Evrysdi has demonstrated consistent clinically meaningful efficacy in adults, children, and babies two months and older and is now approved in 58 countries worldwide
Further presentations included data from studies supporting the efficacy, safety, and sturdiness of gene therapy, SRP-9001, within the treatment of Duchenne dystrophy (DMD)

Roche today announced data from across its growing neuromuscular portfolio at the planet Muscle Society (WMS) Virtual Congress 20 – 24 September 2021. The presentations included additional results from the RAINBOWFISH study, evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA) from birth to 6 weeks aged and data supporting the continued clinical investigation of gene therapy, SRP-9001, in Duchenne dystrophy (DMD)

“These new data for Evrysdi may help extend the potential benefits of this medicine to the youngest SMA patients. Also, the info from SRP-9001 have helped to optimise the planning of the upcoming phase III clinical trial trial for DMD,” said Levi Garraway, M.D., Ph.D, Roche’s Chief medic and Head of worldwide development . “Our goal is to still lead the way in developing transformative medicines for neuromuscular diseases. We are grateful for the partnerships that are helping us to develop new therapies for people impacted by these devastating rare diseases.”

At WMS 2021, data from the continued open label RAINBOWFISH study were presented. Four out of 5 of these treated with Evrysdi for a minimum of 12 months achieved standing and walking independently within the planet Health Organization windows for healthy children. additionally , all five babies maintained the power to swallow and were ready to feed exclusively orally after 12 months of treatment.

Previously reported results showed that babies treated with Evrysdi for a minimum of 12 months achieved Hammersmith Infant Neurological Examination (HINE-2) motor milestones, with 100% (n=5) ready to maintain head control, sitting upright, rolling and crawling.

These data further increase the growing body of evidence supporting Evrysdi’s efficacy during a broad patient population. quite 4,000 patients are treated with Evrysdi in clinical trials, compassionate use, and real-world settings.

In addition, results from Part 1 of Study SRP-9001-102, an ongoing, randomised, double-blind, placebo-controlled clinical test evaluating the security , efficacy, and tolerability of one dose of SRP-9001 in 41 boys with DMD, showed that the study met its primary biological endpoint of change in micro-dystrophin protein expression from baseline. Participants treated with SRP-9001, generally showed a rise in NSAA total score compared to placebo at 48 weeks, although this increase didn’t achieve statistical significance compared thereto of patients who received placebo. the security profile was according to prior studies, with no new safety signals identified.

About Evrysdi® (risdiplam)
Evrysdi may be a survival of efferent neuron 2 (SMN2) splicing modifier designed to treat SMA by increasing production of the survival of efferent neuron (SMN) protein. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement. Evrysdi is run daily reception in liquid form orally or by feeding tube.

The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) approved Evrysdi for the treatment of SMA in adults and youngsters 2 months aged and older. Evrysdi was granted PRIME designation by the EMA in 2018 and Orphan Drug Designation by FDA and EMA in 2017 and 2019, respectively. At this point , Evrysdi has been approved in over 50 countries including the US and Europe and submitted in additional than 30 countries.

About SMA
SMA may be a severe, progressive neuromuscular disease which will be fatal. It affects approximately one in 10,000 babies and is that the leading genetic explanation for infant deathrate . SMA is caused by a mutation of the survival efferent neuron 1 (SMN1) gene, which results in a deficiency of SMN protein. This protein is found throughout the body and is important to the function of nerves that control muscles and movement.

Without it, nerve cells cannot function correctly, resulting in muscle weakness over time. counting on the sort of SMA, an individual’s physical strength and their ability to steer , eat or breathe are often significantly diminished or lost.

About SRP-9001
SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) is an investigational gene therapy designed to deliver the micro-dystrophin-encoding gene on to the skeletal and heart muscle for the targeted production of the micro-dystrophin protein to enable a durable clinical response. Sarepta Therapeutics is liable for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 within the us upon receiving FDA approval. In December 2019, Roche partnered with Sarepta to mix Roche’s global reach, commercial presence, and regulatory expertise to accelerate access to SRP-9001 for patients outside the us .

About DMD
DMD may be a rare X-linked, progressive neuromuscular disease caused by mutations within the DMD gene that disrupts the assembly of functional dystrophin protein, resulting in a loss of muscle function and premature death. DMD is one among the foremost common fatal genetic disorders, affecting approximately one in every 3,500 to 5,000 male births worldwide.

Symptoms usually appear in infants and toddlers, with affected children presenting developmental delays like difficulty walking, climbing stairs or standing from a sitting position. As DMD progresses, muscle weakness involves the arms, trunk, and other areas, meaning patients often require full-time use of a wheelchair in their early teens. Longevity is restricted thanks to cardiac and/or respiratory failure.

About Roche
Roche may be a global pioneer in pharmaceuticals and diagnostics focused on advancing science to enhance people’s lives. The combined strengths of pharmaceuticals and diagnostics, also as growing capabilities within the area of data-driven medical insights help Roche deliver truly personalised healthcare. Roche is functioning with partners across the healthcare sector to supply the simplest look after everyone .

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