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10:11
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Originally posted by Zenox:
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Dear all A biologics license application may be approved on thebasis of a demonstration that the biological product that is the subject of theapplication is “safe, pure, and potent” (42 USC 262(a)(2)(c)(i)(I). Federalregulations provide the following definitions of potency that apply tocell-based drug products: VOTE: Do the available data support the efficacy of remestemcel-L in pediatric patients with steroid-refractory aGVHD? Now consider the following : “FDA has considered single-arm trials to support a marketing approval in instances where there are no available therapies that would be considered standard of care, where the effect of response is presumed to be attributable to the investigational product.4 FDA concluded for ruxolitinib that since the disease is life-threatening, there were no approved therapies, there was no optimal therapy of aGVHD identified, the efficacy endpoint was objective, the activity of the drug was established in other diseases that shared similar pathophysiology as with aGVHD, and there was a substantial safety database, a single-arm trial as the sole basis of efficacy would be acceptable. Further, the Day-28 ORR of 57.1% with a lower 95% CI bound excluding 40% with durability was considered clinically meaningful for patients with SR-aGVHD.5 Lastly, due to the fact that the lowest available strength of ruxolitinib precluded safe treatment in infants and children, the indication was limited to patients 12 years and older.5 There are no drugs approved for treatment of SR-aGVHD in patients less than 12 years old. There are 14 drugs listed in the National Comprehensive Cancer Network (NCCN) guidelines as "suggested" systemic agents for treatment of SR-aGVHD.6 All are stated to have only Category 2A evidence. There was not sufficient data to recommend use of one agent over others.”
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I hope you're right and I hope I'm wrong, but every time someone quotes the Jakafi "precedent" they ignore these words "FDA concluded for ruxolitinib that since ... the activity of the drug was established in other diseases that shared similar pathophysiology as with aGVHD, and ... a single-arm trial as the sole basis of efficacy would be acceptable."
(20min delay)