Genentech’s Evrysdi (Risdiplam) Granted FDA Priority Review for Treatment of Pre-Symptomatic Infants Under 2 Months with Spinal Muscular Atrophy

News on January 25, 2022 /BioValleyBIOON/ -- Roche's Genentech recently announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental New Drug Application (sNDA) for the oral medication Evrysdi® (Chinese trade name: 艾满欣®; generic name: risdiplam) and granted it priority review for the treatment of pre-symptomatic infants under 2 months of age with Spinal Muscular Atrophy (SMA). This sNDA includes interim data from the RAINBOWFISH study, which shows:Most presymptomatic infants treated with Evrysdi achieved key milestones such as sitting, standing, and walking after 12 months of treatment, and maintained their swallowing ability.

Evrysdi aims to treat SMA by increasing and maintaining the production of survival motor neuron (SMN) protein in the central nervous system (CNS) and peripheral tissues. SMN protein is found throughout the body and is crucial for maintaining healthy motor neurons and movement. Currently, Evrysdi'sFDAIndicated for: Treatment of SMA in adults, children, and infants 2 months of age and older.If the sNDA is approved, Evrysdi will become the first drug to treat SMA symptoms in infants before symptom onset at home.

Levi Garraway, Chief Medical Officer and Head of Global Product Development at Genentech, stated: "Treating very young infants with Evrysdi before the onset of SMA symptoms may help them achieve milestones such as standing and walking within the typical timeframe for healthy infants. Expanding treatment options for the youngest members of the SMA community is crucial, and we look forward to collaborating with...FDA"Collaborate to resolve this application."

Preliminary interim data from the RAINBOWFISH study, presented at the World Muscle Society (WMS) 2021 Virtual Congress, showed that in the infants included in the interim efficacy analysis,All (5/5) maintained the ability to swallow and were able to be fully orally fed after 12 months of treatment. Eighty percent (4/5) achieved milestones such as independent standing and walking within the World Health Organization (WHO) healthy children’s window after receiving Evrysdi treatment for at least 12 months. All infants (n=5) reached motor milestones including head control, sitting upright, rolling, and crawling in Section 2 of the Hammersmith Infant Neurological Examination (HINE-2) after 12 months of Evrysdi treatment.

During the interim safety analysis (n=12), no treatment-related serious adverse events were reported in any infant receiving Evrysdi. Four treatment-emergent adverse events were reported, all of which have been resolved or are resolving with continued Evrysdi treatment. The most common adverse events (AEs) were nasal congestion (33%), cough (25%), teething (25%), vomiting (25%), eczema (17%), abdominal pain (17%), diarrhea (17%), gastroenteritis (17%), papule (17%), and fever (17%). The adverse events reflected the age of the infants rather than SMA.

The RAINBOWFISH study is currently enrolling patients, with the latest results to be presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in March 2022.MeetingPublished on.

SMA is a motor neuron disease that causes muscle weakness and progressive loss of motor function. It is an autosomal recessive genetic disorder caused by gene defects, affecting muscles throughout the body. Patients mainly exhibit overall muscle atrophy and weakness, gradually losing various motor functions, including even breathing and swallowing. SMA is the leading cause in infants and toddlers under 2 years old.GeneticsKiller disease, which is a relatively common "rare disease," with an incidence rate of 1:6000-1:10000 in newborns. According to relevant reports, the number of SMA patients in China is currently about 30,000 to 50,000.

Evrysdi was approved by the U.S. in August 2020FDAApproved for the treatment of SMA patients aged ≥2 months, both children and adults. In March 2021, Evrysdi received EU approval for the treatment of patients aged ≥2 months with clinical...DiagnosisFor patients with Type 1/2/3 SMA or 5q SMA carrying 1-4 copies of SMN2.In China, in June 2021, Evrysdi (艾满欣®，Risdiplam Oral Solution) was approved by the National Medical Products Administration for the treatment of SMA patients aged 2 months and above.To date, Evrysdi has been approved in 70 countries and submitted for marketing authorization in an additional 31 countries.

Evrysdi is a survival motor neuron 2 (SMN2) mRNA splicing modifier that treats SMA by increasing the production of survival motor neuron (SMN) protein. SMN protein is found throughout the body and is essential for maintaining healthy motor neurons and movement. Evrysdi can continuously increase and sustain SMN protein levels in both the central nervous system and peripheral tissues.

An increasing number of clinical proofs indicate that SMA is a multi-system disease, and the loss of SMA protein may affect many tissues and cells beyond the central nervous system. After oral administration of risdiplam, it shows systemic distribution and can continuously increase the levels of SMN protein in both the central nervous system and peripheral tissues, which has demonstrated improvement in motor function for patients with Type 1, Type 2, and Type 3 SMA. From keyClinical TrialThe data confirmed that Evrysdi is effective for infants aged 2 months and above, as well as for children and adult patients. It demonstrated clinically meaningful improvements in motor function across SMA patients of different ages and disease severities (including Type 1, Type 2, and Type 3). Infants treated with Evrysdi were able to sit unassisted for at least five seconds, a critical motor milestone unattainable in the natural course of SMA. Additionally, compared to natural history, Evrysdi also improved survival rates without permanent ventilation. (Bioon.com)

Source of Original Text:Genentech’s Evrysdi (risdiplam) Granted FDA Priority Review for Treatment of Pre-Symptomatic Babies Under 2 Months of Age With Spinal Muscular Atrophy (SMA)