SMA's first oral medicine! Roche Evrysdi's application for listing in Japan has been reviewed in China.

In August, risdiplam (brand name: Evrysdi) was approved by the U.S. FDA for the treatment of children 2 months and older and adult SMA patients.
Evrysdi is a liquid preparation that can be delivered at home or by mouth 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 treatment for SMA and the first SMA treatment to be given at home.
Evrysdi is a motor neuron survival gene 2 (SMN2) mRNA shear modifier that treats SMA by increasing the production of motor neuron survival protein (SMN).
is found throughout the body and is essential for maintaining healthy motor neurons and exercise.
in 2 clinical trials, Evrysdi showed clinically significant improvements in motor function in patients with SMA of different ages and disease severity (including type 1, 2, and type 3).
who were treated with Evrysdi were able to sit without support for at least five seconds, a key sporting milestone common in the natural course of SMA disease.
, Evrysdi also increased its survival rate without permanent breathing for 12 and 23 months compared to natural history.
approved by Evrysdi, the FDA also issued Roche-owned Geneneck with a Rare Pediatric Disease Priority Review Certificate (PRV).
as part of its ongoing commitment to SMA patients, Genentek has also submitted listing applications in Brazil, Chile, Indonesia, Russia, South Korea, China (china and Taiwan). Dr. Osamu Okuda, President and Chief Operating Officer of
Chinese and Foreign Pharmaceuticals, said, "As the first oral drug to treat SMA, risdiplam has demonstrated clinically significant therapeutic results for infants and adult patients in two clinical studies of Type 1, Type 2 and Type 3 SMA.
believe that risdiplam will make a significant contribution to SMA treatment, and its efficacy has been proven.
we will strive to obtain regulatory approval for risdiplam in order to provide this new oral therapy to SMA patients in Japan as soon as possible.
" risdiplam' FDA-approved NDA in Japan is based on data from two clinical studies that represent a wide range of real-world SMA populations: the FIREFISH study in infants with symptoms aged 2-7 months, and the SUNFISH study in children and adults ages 2-25.
, the SUNFISH study was the first and only placebo-controlled study to include adult patients with type 2 and type 3 SMA.
-- In the FIREFISH study: (1) Using Bailey's Infant Development Scale 3 (BSID-III), 41% (7/17) of infants treated were able to sit without support for at least 5 seconds.
(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 are able to survive without permanent breathing after at least 23 months of treatment and are 28 months or older (median 32 months; range 28-45 months).
in untreated infant-type SMA natural history, infants cannot sit alone, and only 25 percent are expected to survive without permanent breathing after 14 months of age.
- In the SUNFISH study: (1) measuring the total score of 32 scales (MFM-32) using motion function, children and adults treated with Evrysdi had clinical and statistically significant improvements in motor function at 12 months compared to the placebo group (1.36 points, respectively) 95% CI: 0.61, 2.11? vs -0.19 points (95% CI: -1.22, 0.84); average difference: 1.55 points, p=0.0156).
(2) By improving the upper limb module (RULM) measurement, the upper limb movement function is improved compared to the baseline (difference: 1.59 points, p-0.0028), which is a secondary independent movement function end point of the study.
2 studies, Evrysdi has good efficacy and safety.
most common adverse reactions are fever, diarrhea and rash.
the most common adverse events in infested SMA, in addition to upper respiratory tract infections, pneumonia, constipation and vomiting.
two studies did not find any treatment-related safety findings that led to withdrawal from the study.
chemical structure (Photo: medchemexpress.cn) Evrysdi is an oral liquid whose active drug ingredient, risdiplam, is a motulation modifier for the survival gene 2 (SMN2) of motor neurons, designed to continuously increase and maintain SMN protein levels in the central nervous system and peripheral tissues.
increasing clinical evidence that SMA is a multi-system disease and that the loss of SMA proteins may affect many tissues and cells outside the central nervous system.
the systemic distribution after oral dosing of risdiplam, which continuously increases the level of SMN protein in the central nervous system and peripheral tissues, has been shown to improve the motor function of patients with type 1, 2 and 3 SMA.
as part of a partnership with the SMA Foundation and PTC Therapeutics, Geneneck led the clinical development of Evrysdi.
as part of a large-scale, extensive and robust clinical trial project in the SMA field, Evrysdi is conducting research in more than 450 people.
program covers 2-month-olds to 60-year-olds with different symptoms and motor functions, such as scoliosis or joint contractions, as well as patients who have previously received other SMA therapies.
clinical trials of the drug are designed to represent a wide range of people with real-world SMA disease, with the aim of ensuring that all suitable patients have access to treatment.
, Roche is currently conducting 4 global multi-center clinical studies (SUNFISH (NCT02908685), FIREFISH (NCT02913482), JEWELFISH (NCT0303) 2172, RAINBOWFISH (NCT03779334) to evaluate the efficacy and safety of Evrysdi treatment of all types of SMA (Type 1, Type 2, Type 3) and pre-neonatal symptoms SMA.
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, a genetic defect caused by the normal chromosomal recessive genetic disease, the muscles in the patient's body will be damaged, the patient is mainly manifested in the body muscle atrophy, the body gradually lost all kinds of motor functions, and 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 1:6,000-1:10,000 in newborns.
reported that the number of SMA patients in China is currently about 30,000 to 50,000.
December 2016, the drug Spinraza (nusinersen), developed by Yanjian and partner Ionis, was approved as the world's first drug to treat SMA.
The drug is an anthropomorphic oligonucleotide (ASO) that is delivered directly to the cerebrospinal fluid (CSF) around the spinal cord via intrauterine injection, altering the cutting of the SMN2 pre-messenger RNA (pre-mRNA) and increasing the production of fully functional SMN proteins.
in patients with SMA, insufficient levels of SMN protein led to the deterioration of the function of motor neurons in the spinal cord.
in clinical studies, Spinraza therapy significantly improved the motor performance of SMA patients.
May 2019, onasemnogene abeparvovec, a gene therapy from Novarma, was approved, making it the world's first gene therapy to treat SMA.
the drug through a single, one-time intravenous infusion after 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).
the 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 is named 5q-SMA.
original source: Chugai Files a New Drug application for Risdiplam as The First Oral Drug for Spinal Muscular Atrophy in Japan.