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EU’s Innovative Drug Approval Tally Shows Sharp Decline

The numbers may be down, but Europeans will in future be able to gain access to a number of highly innovative medicines, including several products for hard-to-treat cancers, a gene therapy for severe and moderately severe hemophilia B, and two vaccines against disease caused by respiratory syncytial virus.

Europe Approvals

UniQure Downsizes Gene Therapy Ambitions, Pins Hopes On Huntington’s Data

Cutbacks to its workforce and early pipeline could help UniQure focus on core assets – but upcoming readouts to Huntington’s disease gene therapy will be key for investors.

Commercial Companies

UniQure Looks For Clear Signal From Huntington’s Disease Gene Therapy

After recent landmark approvals in other neurodegenerative diseases such as Alzheimer’s and amyotrophic lateral sclerosis, hopes are rising that Huntington’s disease could be next to reach a breakthrough.

Neurology Clinical Trials

No AdComm Puts Sarepta On Path To Blockbuster Success With Duchenne Gene Therapy

The US FDA’s waiving of an advisory committee is another promising signal that Sarepta's Duchenne gene therapy SRP-9001 could be approved on 29 May and set it up to become a bigger earner than Novartis’s Zolgensma.

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