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Acer’s ‘Misunderstood Drugs’ Strategy Faces Regulatory Hurdles

A Regulatory Case-Study

 
• By Bridget Silverman

Acer repurposes drugs with available data (like a 40-year-old European antihypertensive) into rare disease treatments (like Edsivo for vascular Ehlers-Danlos syndrome), but approval remains elusive, with a CRL for ACER-001 just as Edsivo is poised to enter a new clinical trial, armed with a new breakthrough designation. The Pink Sheet delves into regulatory history and strategy with CEO Chris Schelling.

Artery
Ehlers-Danlos is a group of connective tissue disorders that can affect many parts of the body, including blood vessels. • Source: Shutterstock

With a US complete response letter for urea cycle disorder therapy ACER-001, rare disease specialty company Acer Therapeutics Inc. holds CRLs for both of the 505(b)(2) new drug applications it has filed – a reminder of the regulatory challenges that can remain for “de-risked” drug candidates with clinical experience in other fields and formulations.

Acer Therapeutics is focused on finding “drugs that were misunderstood” in their original development programs but could have a new life treating serious orphan diseases, CEO Chris Schelling explained in...

Insights And Ideas From Acer’s Experience
  • Manufacturing issues often trip up applications using the 505(b)(2) route.
  • Acer’s first NDA for Edsivo used data from a trial already conducted by a Paris-based university hospital,...

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