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EMA Grants First Two PRIME Designations In 2024 To Ultragenyx & Sangamo

 
• By Neena Brizmohun

Ultragenyx’s antisense oligonucleotide for treating Angelman syndrome and Sangamo’s gene therapy for Fabry disease are the latest products to make it onto the European Medicines Agency’s priority medicines scheme.

The PRIME scheme aims to help developers of medicines for unmet medical needs • Source: Shutterstock

GTX-102, Ultragenyx Pharmaceutical’s drug candidate for treating Angelman syndrome, and isaralgagene civaparvovec, Sangamo Therapeutics’ investigational gene therapy for Fabry disease, have become the first two products to secure a place on the European Medicines Agency’s PRIME (priority medicines) scheme in 2024.

Both products were accepted onto the scheme for speeding up the development of medicines for unmet medical needs in

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Pharma’s Protests Fall Flat As UK Locks In High Rebate Rates

 
• By Neena Brizmohun

Despite pushback by industry, the government is standing by its new statutory scheme rebate rates for branded medicines, including one that doubles the rate that companies must repay on the sales of newer products to the National Health Service to 31.3%.

EUCOPE Wants Simpler Manufacturing Rules And Framework For Combined Trials Under EU Biotech Act

 
• By Eliza Slawther

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UK MHRA Helps Sponsors Prepare For ‘World’s First’ Decentralized Manufacturing Framework

 
• By Neena Brizmohun

The UK drug regulator, the MHRA, is set to launch its decentralized manufacturing framework in July, and has issued a series of guidance documents that it believes will help companies and also be of value to other countries that are considering how best to regulate the fledging sector.

More from Geography

EUCOPE Wants Simpler Manufacturing Rules And Framework For Combined Trials Under EU Biotech Act

 
• By Eliza Slawther

EU authorities must “take advantage” of the upcoming Biotech Act to reassess the bloc’s policies and “offset existing challenges” for small and midsized enterprises, trade association EUCOPE has said.

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Citeline Japan Awards 2025 - Enter Now!

 
• By Ian Haydock and Lisa Takagi

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