Risdiplam (Evrysdi): A Novel Oral SMN2 Splicing Modifier for the Treatment of Spinal Muscular Atrophy

risdiplam

CAS: 1825352-65-5

Development Company:Genentech

Indications: Spinal Muscular Atrophy(SMA)

Target:SMN2

Time to Market:2020Year8Month7DayJapan Launch，2020Year4Month24Day，National Medical Products Administration Officially Accepts Treatment for Spinal Muscular Atrophy(SMA)DrugrisdiplamMarketing Authorization Application.2018Year12Month19Day - Swiss pharmaceutical giant Roche(Roche)Recently announced,European Medicines Agency(EMA)Grantedrisdiplam(RG7916)Treatment of Spinal Muscular Atrophy(SMA)Priority Drug Eligibility(PRIME)。

Dosage Form: Oral Liquid,0.75MG/ML

Clinical

In the name ofFIREFISHIn the clinical trial,1TypeSMAThe patient received different doses ofrisdiplamTreatment. Day of the trial1Part of the dose escalation study, while in the trial's2Partially, patients receive the expected therapeutic dose ofrisdiplamTreatment. The2Partial trial results indicate that treatment was administered.12Months later,17Among the patients receiving treatment,7Name of the baby (41.2%) can sit up independently for more than5Seconds,9Name of the baby (52.9%) can keep the head straight,1Name of the baby (5.9%) Reach the milestone of motor ability to stand.

InSUNFISHIn clinical trials, the age is2-25Years between2Type and3TypeSMAThe patient receivedrisdiplamTreatment. This trial is also divided into two parts, the first1Part is a dose-escalation study. After12months of treatment, the patientSMNThe average protein expression level increased1Times or more. UseMFM32Scale for Participants in the1The assessment of motor ability in some studies showed that,58%Patient'sMFM32The score improved by at least compared to the baseline3Points.

Original Research Route

Ref:US9969754

Compound Patent

WO2015173181，ApplicantROCHE，Filing Date2015Year5Month11Day. Same ClanCN106459092 (B)，Authorization Time2019Year10Month15 Day.