As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...

The FDA has accepted Sobi’s sBLA for Gamifant for haemophagocytic lymphohistiocytosis (HLH)/MAS associated with Still’s ...

Alltrna’s CEO Michelle Warner said that the approach allows for basket trials that can group patients with different diseases ...

New EU Clinical Trial Regulations simplify international trials while offering the means to counter drug shortages for trials ...

The MSSG solidarity mechanism enables medicine sharing between EU states, with companies now required to report on drug ...

Vietnam is aiming to attract overseas investment to encourage domestic drug development through the amended 2016 Law on ...

The FDA has granted fast track designation to Pyxis oncology’s PYX-201 for the treatment of adults with R/M HNSCC.

Deal numbers dropped in January this year compared to last, with outbound deals trumping inbound transactions.

Pfizer has ended the global commercialisation of its haemophilia B (factor IX deficiency) gene therapy Beqvez, citing soft ...

Zevra has sold its US Food and Drug Administration (FDA) priority review voucher (PRV) for $150m to an undisclosed party.

A survey has shown that pharma companies have low confidence their rare disease medicines can launch in the UK.

Medigene and EpimAb Biotherapeutics have signed a co-development agreement involving T cell receptor (TCR)-guided T cell ...