Spinal muscular dystrophy (SMA) first oral medication! Roche Evrysdi has been approved for listing by the FDA and has submitted an application for listing in China!

!--ewebeditor:page title"--August 08, 2020 // -- Roche's GeneNek recently announced that the U.S. Food and Drug Administration (FDA) has approved Evrysdi (risdiplam) for the treatment of children and adults aged 2 months and older with spinal muscular dystrophy (SMA).
Evrysdi is a liquid preparation that can be given at home by oral or feeding tube once a day and can be used to treat infants, children, adolescents, and adult patients of all types (type 1, type 2, type 3) SMA.
it's worth noting that Evrysdi was the first oral therapy to treat SMA and the first SMA therapy to be delivered at home.
Evrysdi is a motor neuron survival gene 2 (SMN2) mRNA clipping modifier that treats SMA by increasing the production of motor neuron survival protein (SMN).
SMN protein is present throughout the body and is essential for maintaining healthy motor neurons and movement.
2 clinical trials, Evrysdi showed clinically significant improvements in motor function in patients with SMA at different ages and severity of the disease, including type 1, type 2, type 3.
who have been treated with Evrysdi are able to sit for at least five seconds without support, a key sporting milestone common in the natural course of SMA disease.
addition, Evrysdi's survival rate without permanent breathing increased by 12 and 23 months compared to natural history.
regulatory aspects, the FDA granted Evrysdi rapid-track eligibility (FTD) and Priority Review (PR) for listing applications.
, the FDA also granted Evrysdi orphan drug qualification (ODD) for the treatment of SMA, a program designed to provide incentives to help and encourage drug development for rare diseases.
Evrysdi was approved, the FDA also issued Gene Tek to a Rare Pediatric Disease Priority Review Certificate (PRV).
as part of its ongoing commitment to SMA patients, Genentic has also submitted applications for listing in Brazil, Chile, Indonesia, Russia, South Korea, China (mainland China and Taiwan) and is about to submit an application to the European Medicines Agency (EMA). Dr. Levi Garraway, Chief Medical Officer and Head of Global Product Development at
Roche, said, "Given that most SMA patients in the United States remain untreated, we believe that Evrysdi, with its good clinical performance and oral medication, will bring meaningful benefits to many patients with this rare neurological disorder."
and determination of the SMA community has always inspired us as we developed the first SMA oral therapy, so today we celebrate our collective achievements with them.
" SMA Boys (pictured: drpgx.com) is based on data from two clinical studies that represent a wide range of real-world SMA populations: the FIREFISH study in infants with symptomasis at 2-7 months of age and the FUNFISH study in children and adults aged 2-25 years.
, the SUNFISH study was the first and only placebo-controlled study involving adult patients with type 2 and 3 SMA.
- In the FIREFISH study: (1) Measured using the third edition of the Bailey Infant Development Scale (BSID-III), 41% (7/17) of infants receiving treatment were able to sit for at least 5 seconds without support.
(2) 90% (19/21) of infants are able to survive without permanent breathing at 12 months of treatment and are 15 months or older.
(3) 81% (17/21) of infants were able to survive without permanent breathing after at least 23 months of treatment, aged 28 months or more (median 32 months; range 28-45 months).
in the natural history of untreated infant-type SMA, babies cannot sit alone, and only 25 percent of babies are expected to survive without permanent breathing after 14 months of age.
- In the SUNFISH study: (1) Total score measurement of 32 gauges (MFM-32) using motor function was clinically significant and statistically significant for children and adults treated with Evrysdi at 12 months compared to the placebo group (1.36 points, respectively) (95%CI:0.61, 2.11) vs .19 points (95%CI:-1.22, 0.84); Average difference: 1.55 points, p.0156).
(2) improved upper limb movement function compared to the baseline (difference: 1.59 points, p-0.0028), which is a secondary independent motor function end of the study.
two studies, Evrysdi had good efficacy and safety.
most common adverse reactions are fever, diarrhea and rash.
are similar to the most common adverse events in infant-onset SMA, in addition to upper respiratory tract infections, pneumonia, constipation and vomiting.
two studies found no findings of treatment-related safety that led to withdrawal from the study. kenneth Hobby, president of
Cure SMA, said: "In the lifetime of SMA patients, many people may lose the ability to perform key actions, which can affect their ability to participate independently in all aspects of their daily lives and even change their lives.
Evrysdi's approval is a milestone that our SMA community is looking forward to.
we appreciate Genenta's commitment to fully reflecting the real-world SMA population in clinical trial programs and developing a treatment that can be used at home.
" risdiplam chemical structure (Photo Source: medchemexpress.cn) Evrysdi is an oral liquid, and its active drug ingredient, risdiplam, is a motor neuron survival gene 2 (SMN2) shear modifier designed to continuously increase and maintain SMN protein levels in the central nervous system and peri-peri-tissue.
a growing body of clinical evidence suggests that SMA is a multisystal disease and that the loss of SMA proteins can affect many tissues and cells outside the central nervous system.
systemic distribution after oral application of risdiplam, which continuously increases SMN protein levels in the central nervous system and outer tissues, has been shown to improve motor function in patients with type 1, type 2 and type 3 SMA.
!--/ewebeditor:page-!--ewebeditor:page title"--as part of a partnership with the SMA Foundation and PTC Therapeutics, Genetek led the clinical development of Evrysdi.
Evrysdi is being studied by more than 450 people as part of a large-scale, extensive and robust clinical trial in the SMA field.
the program covers 2-month-olds to 60-year-olds who have different symptoms and motor functions, such as scoliosis or joint contractions, as well as patients who have previously received other SMA treatments.
clinical trials of the drug are designed to represent a wide range of real-world SMA patients, with the aim of ensuring that all suitable patients have access to treatment.
currently, Roche is conducting four global multi-center clinical studies (SUNFISH (NCT02908685), FIREFISH (NCT02913482) and JEWELFISH (NCT0303) 2172, RAINBOWFISH (NCT03779334) to evaluate the efficacy and safety of all types of Evrysdi treatment (type 1, type 2, type 3) SMA and pre-symptom sMA in newborns.
Spinraza: The world's first SMA treatment drug, has been approved in China SMA is a motor neurone disease that causes muscle weakness and atrophy, the disease is a genetic defect caused by autosomal recessive genetic disease, the patient's body will cause damage to the muscles under the body, the patient mainly manifests as the whole body muscle atrophy, the body gradually loses all kinds of motor functions, even breathing and swallowing.
SMA is the number one genetic disease killer among infants and young children under 2 years of age, a relatively common "rare disease" with a prevalence rate of 1:6000-1:10,000 in newborns.
according to relevant reports, the number of SMA patients in China is currently about 3-5 million.
December 2016, the drug Spinraza (nusinersen), developed by Yan Jian and partner Ionis, was approved, becoming the world's first drug to treat SMA.
The drug is an antiseemtic oligonucleotide (ASO) that is injected into the intrauterine to deliver the drug directly to the cerebrospinal fluid (CSF) around the spinal cord, altering the scission of the pre-MESSENGER RNA (pre-mRNA) of SMN2 to increase the production of the fully functional SMN protein.
in SMA patients, insufficient levels of SMN protein lead to degeneration of motor neuron function in the spinal cord.
in clinical studies, Spinraza therapy significantly improved motor performance in SMA patients.
May 2019, Novarhua's gene therapy, Onasemnogene Abeparvovec, was approved as the world's first gene therapy to treat SMA.
the drug through a single, one-time intravenous infusion after the continuous expression of SMN protein to stop the disease process, can solve the underlying causes of SMA, is expected to improve the quality of life of patients in the long term.
the Chinese market, Spinraza was approved at the end of February 2019 for the treatment of patients with 5q spinal muscular dystrophy (5q-SMA).
approval, making Spinraza the first drug to treat SMA in the Chinese market.
5q-SMA is the most common type of SMA, accounting for about 95% of all SMA cases, which is caused by mutations in the SMN1 (motor neuron survival protein 1) gene on chromosome 5 and hence the name 5q-SMA.
source: FDA Approves Genentech's Evrysdi (risdiplam) for Treatment of Spinal Musc Atrophy (SMA) in Adults and Children 2 Months and Older !--/ewebeditor:page.