Authored by Karl Simpson
This week sees the World Orphan Drug Congress in Geneva, Switzerland. After a successful WODC event back in April in Washington, we’re looking forward to a very engaging conference with some of the industry’s most illustrious rare disease experts and leaders. Liftstream is sponsoring this event this year, having also sponsored and supported the 2013 US event too.
It has been a big year for orphan drugs and rare diseases. The broader pharma industry has really started to focus on this area of orphan drug development. We have seen companies like Aegerion and NPS Pharmaceuticals begin to see the initial successes of their respective launches for Juxtapid and Gattex. The Duchenne Muscular Dystrophy diseases area has been very prominent with lead development projects from Sarepta, Prosensa/GSK, PTC Therapeutics and Summit Plc all making significant advances, as well as new funding or operational capability.
There have been many rumours circulating about acquisitions and Shire finally stepped forward with a significant deal to acquire ViroPharma only yesterday. There have also been a number of Orphan Drug companies which have achieved IPOs this year as the public markets funding have opened to biotechs again. It seems the biotech asset class is back in favour and more specifically rare diseases is attracting public and private investors. Venture Capital firms have raised new funds dedicated to rare diseases, while VC powerhouses like David Mott’s NEA have also been active in this category, establishing a ‘rare diseases accelerator’ in Cydan.
All of these very dynamic changes have really created a strong interest in the rare disease market. For Liftstream, we’ve been working in orphan drugs and rare diseases since we started the company nearly a decade ago, so it is good to look forward to this upcoming event knowing there is so very much to discuss. We look forward to seeing you there.