GSK Takes Rare Diseases Option from Prosensa

01 Nov 2009

• By Melanie Senior

GlaxoSmithKline's tie-up with Dutch biotech Prosensa around four RNA-modulating therapeutics for Duchenne Muscular Dystrophy, a rare genetic disorder, provides recent evidence that even the largest Big Pharma are embracing the most niche of specialist disease indications.

There's no denying that even the largest Big Pharmas are embracing the most niche of specialist disease indications. GlaxoSmithKline PLC's October 13 tie-up with Dutch biotech Prosensa Holding BV around four RNA-modulating therapeutics for Duchenne muscular dystrophy (DMD), a rare genetic disorder that affects one in 3,500 newborn boys, provides recent evidence. [See Deal]

The two-part deal sees GSK paying $25 million (£16 million) up front for a worldwide license to Prosensa's lead Duchenne...