Roche releases new data from two-year study of risdiplam in the treatment of infants, children and adults with SMA

Guide: Rocherisdiplam has obtained positive data on patients who have used both therapies in the treatment, or will further increase risdiplam's advantages and competitiveness in the market, Roche's Genetek released positive data on two years of treatment for risdiplam in patients with type 2-2 or type 3 spinal muscular dystrophy (SMA) in part 1 of SUNFISHThe results of the exploratory efficacy analysis showed that the movement function of patients was significantly improved after 24 months of risdiplam treatment compared to natural history dataIn addition, preliminary data from 12 months of study JEWELFISH, a trial of various types of SMA patients who had previously been treated, showed that levels of the surviving motor neuron (SMN) protein increased rapidly and continuously after treatment with risdiplamSUNFISH is a global study of SMA children and adult patients (n-231) in two partsThe first part of the study (n-51) focused on determining the dose, including a wide range of patients, from unable to sit down to being able to walk, as well as patients with scoliosis or joint contractionsThis part of the exploratory efficacy analysis uses the Motion Function Measurement (MFM) scale to assess the patient's motor function's weighted analysis of the data compared to a reliable natural historical comparator queue showed that patients treated with risdiplam had a significant overall change in MFM compared to the baseline at the 24th month (3.99 points difference (95% CI: 2.34, 5.65) p .0001)Compared to placebo, the total MFM-32 score of patients treated with risdiplam also improved significantly compared to the baseline (average difference of 1.55 points; p-0.0156)Even small changes in motor function can make meaningful differences in patients' daily livesresults also showed that after four weeks of risdiplam treatment, the level of SMN protein in patients' blood was on average tripled and lasted at least 24 monthsThe results were consistent with the results of a 12-month trial in patients with non-bed-in-bed in the second part of the previously reported SUNFISH key studythe most common adverse events in the first part of theSUNFISH study were fever (55%), cough (35%), vomiting (33%), upper respiratory tract infections (31%), colds (nasopharyngitis, 24%) and sore throats (pain in the mouth, 22%)The most common serious adverse event in 51 patients treated with risdiplam was pneumoniaSo far there have been no treatment-related safety findings that could lead to discontinuationIn addition , the study of THE JEWELFISH study was completed to assess the safety and pharmacodynamic data of the use of risdiplam in Previously Treated SMA patients Of these, 76 had received nusinersen (SPINRAZA), 14 had received onasemgene abeparvovec (Zolgensma) and the remaining 83 patients had received rislamdip After 12 months of treatment with risdiplam, the patient's SMN protein was observed to have tripled relative to the baseline level (n?18) early safety assessments showed consistent safety compared to untreated patients The most common adverse events were upper respiratory tract infections (13 per cent), headache (12 per cent), fever (8 per cent), diarrhoea (8 per cent), nasopharyngitis (7 per cent) and nausea (7 per cent) There are no drug-related safety findings that can lead to withdrawal from the JEWELFISH trial, and the overall adverse event characteristics are similar to those in patients who did not receive SMA-targeted treatment in the risdiplam trial SMA is a hereditary, sexualneuromuscular disease that can lead to devastating muscle atrophy and disease-related complications It is the most common genetic cause of infant death and one of the most common rare diseases, affecting about one in every 11,000 babies Patients with type 1 SMA usually do not live beyond 2 years of age, but patients with mild type 2 and type 3 can have longer survival times However, the mild form of the disease is still associated with mobility, respiratory infections and death there are a number of sashimi treatments available for SMA, there is still a huge demand in the market in this area Spinraza, the world's first approved SMA treatment, generated total sales of $2,097 million in 2019, up 22% year-on-year, with treatment costs of $750,000 per year The second SMA treatment, Zolgensma, went on sale in May 2019, with sales of $361 million in the first three quarters Rocherisdiplam has obtained positive data on patients who have used both therapies in treatment, or will further increase risdiplam's advantages and competitiveness in the market Source: Genentech Announcs 2-Year Risdiplam Data From SUNFISH and New Data From FROM JEWELFISH in Infants, Children and Adults With Spinal Spinal Atrophy (SMA)