Efficacy Hard To Prove In Orphan Diseases; Dyax Panel Questions Standards
One central theme of the recent advisory panel review for Dyax's hereditary angioedema therapy Kalbitor (ecallantide) was awareness that the challenges of studying an orphan disease make it difficult to provide conclusive evidence for a drug candidate
You may also be interested in...
FDA is acknowledging that clinical benefit for patients with rare conditions may best be verified through post-marketing trials, given the difficulty of conducting clinical trials in those populations
The hits just keep on coming for Genzyme. On a March 2 call with investors, the firm had to announce not just a "complete response" letter for its 2000-liter scale-up of Myozyme (which will be called Lumizyme for the larger production scale), but also that its Allston Landing manufacturing site had received a 483 report from FDA and a subsequent warning letter
Despite receiving a “complete response” letter and manufacturing warning letter on the same day, Genzyme remains upbeat about the likelihood of approval for its 2,000L scale-up of Myozyme.