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FDA hits Sarepta with liver warning labelling for its DMD drug Elevidys
As well as the new safety warnings for the Duchenne muscular dystrophy gene therapy, the biopharma company also faced a new, more restrictive licence for the treatment.
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Sarepta Therapeutics
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Vinay Prasad returns to lead vaccines and cell and gene therapy at the FDA
CBER head makes surprise comeback just weeks after his abrupt resignation.
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Amid FDA turmoil Sarepta’s gene therapy worries begin to ease
But the impact of George Tidmarsh’s CBER appointment after Vinay Prasad’s abrupt resignation remains to be seen.
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Gene therapy approved for Duchenne muscular dystrophy
The first approved gene therapy for certain paediatric Duchenne muscular dystrophy (DMD) patients leads to production of the Elevidys micro-dystrophin protein.
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Oligonucleotide synthesis market to reach $16.7 billion by 2027
Ongoing clinical trials for oligonucleotide-based therapies will propel the oligonucleotide synthesis market to reach $16.7 billion by 2027, states a new report.
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Complete response letter handed to Sarepta Therapeutics
The pharmaceutical company has received a complete response letter for its golodirsen injection, which had been submitted for accelerated approval.
