FDA grants Roche's preferred review
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Last Update: 2020-06-07
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Source: Internet
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Author: User
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, Roche announced that the u.SFDA(http://has accepted and qualified for priority review ofnew drug(http://About Risdiplam
Risdiplam is an mRNA shear modifier for the SMN2 gene for spinal muscular dystrophy (SMA)Risdiplam is designed to increase and maintain SMN protein levels throughout the central nervous system and peripheral tissues of the bodyThe FDA is expected to make a decision on approval by May 24, 2020previously, the FDA granted the risdiplam orphan drug eligibility in January 2017 and the fast-track qualification in April 2017If approved, risdiplam will be the first oral drug to be used to treat SMA patientsThe currently listed SMA treatmentdrug(http://the gene therapy Zolgensma of Biogen's Spinraza and NovartisRisdiplam's NDA application is based on key research findings from FIREFISH and SUNFISHFIREFISH is a key, single-armtest(http://to evaluate risdiplam in 41 SMA infantsSUNFISH is a two-blind, placebo-controlled, key clinical trial conducted in children and young adults (2-25 years) of type 2 or type 3 SMARisdiplam showed positive clinical efficacy in both trialsTo date, no therapeutic-related safety incidents have been observed in any risdiplam trialthat that led to the withdrawal of the study
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