Challenge:
A biotech developing a therapy for a rare metabolic bone disease faced regulatory misalignment with FDA pain guidelines, risking costly dual Phase 3 trials.
What We did:
Efficus Health crafted an alternative regulatory pathway, engaged directly with the FDA, and supported key study documents (protocol, SAP, IB, ICF, etc.). We brought in our pain-management and clinical development expertise to strengthen arguments and ensure scientific rigor in all submissions.
Outcome:
FDA agreed to one Phase 3 trial instead of two, saving the client millions and accelerating development. These savings gave the biotech critical momentum to advance its pipeline in a highly resource-constrained environment.
“Thanks for the hours, expertise, and effort to get us where we currently are – we wouldn’t be here without you! Your partnership on regulatory strategy and hands-on support with study documents saved us valuable time. You were truly a supportive team member at every step; your dedication will help us to move forward and bring hope to the people with this rare painful condition for which there are no approved therapies.”
Gail Cawkwell | Chief Medical Officer | Ambros Therapeutics, Inc.