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 ...

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

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

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

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 ...

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

Eli Lilly has announced plans to expand its domestic medicine production in the US with four new pharmaceutical manufacturing ...

Last month, the NICE endorsed Casgevy for use in the NHS under a managed access scheme to treat sickle cell disease.

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