Retrophin presentation

Pursuing Accelerated Approval for Rare Disease Therapeutics

In Clinical Development, Orphan Drug Congress, Orphan Drugs Live by Karina Kusova

Alvin Shih, MD, EVP and Head of R&D, Retrophin joined us at the World Orphan Drug Congress USA 2016 to present on Pursuing Accelerated Approval for Rare Disease Therapeutics: Sparsentan Experience and Lessons Learned. 

Download Alvin’s presentation here >>

The Accelerated Approval pathway was put forward in response to the AIDS crisis by the FDA in 1992. It’s purpose was to speed approval for treatments for disease with serious unmet medical needs. Of course, this is regularly the situation when dicusssing rare disease. The pathway allows the FDA to approve a treatment based on an efficacy evaluation using a surrogate endpoint that is “reasonably likely to predict clinical benefit”. Accelerated Approval in rare disease development is vital as diseases often carry a high degree of unmet needs, where the patient population is small thus clinical data is sparse.

“In general, the more rare and severe the disease/condition, the greater the need for regulatory flexibility and access to the AA pathway.”

Download Alvin’s presentation here for more information on:

The Sparsentan Case Study

  • Sparsentan for the Treatment of Focal Segmental Glomerulosclerosis (FSGS)
  • Sparsentan–Dual Mechanism of Action
  • SparsentanPhase 2 Study –DUET

Accelerated Approval

  • What is accelerated approval?
  • Success stories: Using AA and biomarkers in rare disease drug development
  • What makes for a good biomarker?
  • Making the case for proteinuria as a surrogate endpoint
  • ETA receptor antagonists have shown reduction of proteinuria in various clinical settings
  • Recent advancements on the regulatory front

Find out more about the regulatory pathways in Orphan Drug Research by joining us at the World Orphan Drug Congress USA 2017. For example, we have a fantastic Keynote Panel on Prescription Drug User Fee Act (PDUFA VI) and 21ST Century Cures – recent and pending legislative efforts to make drugs better for patients.

World Orphan Drug Congress USA 2017