Rare Disease

Novartis licenses small molecule treatment for a genetic ultra-rare disease

15-Aug-2019 By Maggie Lynch

Novartis enters a licensing and development agreement with Pharming group for ultra-rare disease treatment for more than $20m.

FDA gives a nod to Turalio as the first treatment for rare joint tumor

08-Aug-2019 By Vassia Barba

Daiichi Sankyo’s Turalio becomes the first FDA-approved TGCT treatment, after receiving breakthrough therapy, priority review and orphan drug designation.

Ipsen adds to rare disease portfolio with $1.3bn Clementia deal

26-Feb-2019 By Maggie Lynch

Ipsen acquires Clementia and takes on late-stage clinical asset for the treatment of rare bone disorders, in its largest transaction to date.

Moderna opens manufacturing site in Massachussets to advance its mRNA pipeline

23-Jul-2018 By Maggie Lynch

Moderna Therapeutics has announced the opening of a state-of-the-art manufacturing site in Norwood, Massachusetts, built to advance their work in the messenger RNA (mRNA) platform, after announcing the plans for the site in 2016.

Policy continuity needed to speed up orphan drug development

23-Jun-2003

While the procedure for designating drugs for orphan diseases has
improved dramatically in the EU, further progress must be made,
says EU biotech group