Facet Helps Small Biologics Company Secure Breakthrough Designation on Phase 2 Data

Facet assisted a sponsor in successfully obtaining Breakthrough Therapy Designation for their gene therapy product. In conjunction with the Breakthrough Therapy Designation (BTD), Facet helped the company discuss the results of a Phase 2 study with FDA in a formal meeting.  Subsequently, Facet worked with the company to develop a proposal for the submission of a BLA based on the results of the Phase 2 study as pivotal and multiple ex-US clinical studies as supportive of the efficacy and safety of the product. If successful, the plan will allow the company to save several years of development time and seek approval for the product for treatment of a serious unmet medical need.

Facet Helps Company Establish Strategic Plan to Increase Product Sales

Facet helped a small commercial-stage company evaluate issues associated with sluggish market uptake of their recently approved product. Through a careful landscape and market analysis, Facet and the company identified several limitations of the current product distribution model and brainstormed alternatives. Then, Facet pressure tested each alternative by contacting medical practices, insurance companies, and pharmacies to evaluate the market viability of each option. Plus, Facet evaluated the regulatory implications of each approach, the tactical considerations for changing the distribution model, and the estimated regulatory costs associated with each approach. Facet also recommended the company seek federal and local government feedback and legal counsel prior to making their final decision. Ultimately, Facet provided the company with rank-ordered recommendations based on estimated probabilities of overall success. This integrated approach was instrumental in understanding the multidimensional nature of the product distribution issue and provided the company with actionable guidance to increase product sales, physician awareness, and patient access.