Friday, December 20, 2013

Alexion Pharmaceuticals, cPMP and the FDA's Breakthrough Designation

This post is really more about the new accelerated pathway that drugs like Sofosbuvir and Abbvie's HCV combination therapy are or were developed through. Here are the first two paragraphs. Read the complete post only at Seeking Alpha here.
What it takes to be a winner
Along with Alexion Pharmaceuticals' (ALXN) ALXN1101, more than 30 "Breakthrough Therapy" designations have been granted since the new accelerated approval pathway's inception. As of December 6, 2013, just three (PDF) have resulted in an accelerated approval. They are Genentech's Obinutuzumab, Pharmacyclics' (PCYC) Ibrutinib, and Gilead Science's (GILD) Sofosbuvir. As a potential Alexion investor, I'd like to take a closer look at the winners and the designation's criteria in order to assess the likelihood of ALXN1101's accelerated approval.
Reasons for another expedited pathway
Expedited pathways for life-threatening illnesses lacking available, effective treatments are hardly a recent development at the FDA. However, for patients clearly willing to trade certainty for speed, clinical development requirements were still viewed as far beyond what should be necessary. When a targeted therapy achieves its intended effect on its intended human subjects during early stage clinical trials, traditional late phase trials that last several years seems criminally excessive. The trouble was that prior to the passage of the FDA Safety and Innovation Act of 2012 ((FDASIA)), the requirements for expediting development of clearly effective drugs wasn't as clearly defined as it needed to be.
Again, you can read the complete post here.