Roche's new drug Gazyva treats lupus nephritis CRR up to 40%

November 11, Roche announced that its small-molecule drug for SMA, Risdiplam and CD20 monoantigen Gazyva, had achieved positive results in phase III SUNFISH studies and Phase II NOBILITY studies, respectively. The former is mainly aimed at patients with type 2/3 spinal muscular dystrophy (SMA) aged 2-25 years, while the latter is mainly aimed at patients with lupus nephritis.I. The new oral small molecule SMA drug risdiplam Roch developed risdiplam is an oral drug that can sustain the increase and maintenance of SMN protein levels throughout the central nervous system and tissues around the body. Currently, risdiplam is evaluating the effectiveness and safety of treatment for SMA patients aged 0-60 years, including those who have received SMA targeted therapy.The SUNFISH study is a double-blind, placebo-controlled critical Phase III study, divided into two phases. Phase 1 trials (n-51) were mainly used to determine the dose of phase 2 trials, while Phase 2 trials (n-180) assessed the motor function of SMA patients with MFM-32. The results showed that risdiplam significantly improved the exercise function of patients compared to placebo, and no serious side effects were found. Details of the trial will be presented at an upcoming medical conference.SMA is a motor neurone disease caused by a genetic defect in the SMN1 gene that encodes SMN, a protein necessary for motor neurons to survive, which eventually leads to the death of motor neurons, leading to sexual paralysis in patients. The incidence of SMA is about 1/10000-1/6000 and is the main genetic cause of infant mortality. Depending on the age of onset and severity of muscle weakness, SMA is divided into type I, II, III and type IV. type and prenatal morbidity and type 0 of death within a few weeks.The SMA treatments currently approved for the market are Baijian Spinraza and Novarma gene therapy Zolgensma. Spinraza, which is used for type II SMA patients of all ages through intra-intra-intransibal dosing, had sales of $1,553 million in the first three quarters of 2019, while Zolgensma was approved for use in patients with type I SMA under the age of two, selling for $175 million in four months.Both drugs are sky-high, with Spinraza's treatment costing $750,000 in the first year and halving to $375,000 the next. Zolgensma can fix defective genes to cure disease, requiring only one treatment and priced at $2.1 million.2. Gazyva significantly improves the full kidney response rate in patients with lupus nephritis Gazyva is the first glyco-based modified monoanti, considered to be the next generation of anti-CD20 monoanti. Adaptations that have been approved so far are chronic lymphocytic leukemia and figular lymphoma. In 2018, the drug's global sales were CHF 390 million.The results of the NoBILITY II study, published in the paper, showed that 76 weeks of Gazyva (obinutuzumab) treatment compared to placebo significantly increased the full kidney response rate (40% vs. 18%) in patients with lupus nephritis, reaching the main endpoint of the study. In terms of safety, the rates of serious adverse events in the Gazyva and placebo groups were 24 per cent and 29 per cent, respectively, and the rates of serious infections were 6 per cent and 18 per cent, respectively.
(
Rubik's Cube
)