28 February 2025
28 February 2025
A survey has shown that pharma companies have low confidence their rare disease medicines can launch in the UK.
With issues in therapy delivery and a barren trial landscape, short-term chances for a commercial galactosemia treatment are low.
Deal numbers dropped in January this year compared to last, with outbound deals trumping inbound transactions.
New EU Clinical Trial Regulations simplify international trials while offering the means to counter drug shortages for trials in Europe.
The company's exit from the gene therapy space has reduced the competition for eligible interested patients.
Zevra has sold its US Food and Drug Administration (FDA) priority review voucher (PRV) for $150m to an undisclosed party.
The MSSG solidarity mechanism enables medicine sharing between EU states, with companies now required to report on drug stocks.
The final result of the COSMIC-313 trial is not the only disappointment for Exelixis.
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