By Anna Rose Welch, Director, Cell & Gene Collaborative
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The first time I summited one of the Adirondack High Peaks in the middle of the winter, I did it on all fours because I was afraid the wind was going to blow me out into the void. Even though there was little to see in terms of a landscape beyond endless white in every direction, I will never forget (nor could I ever adequately describe) the size of the clouds billowing towards me. No doubt, you have had similar experiences that were awe-inspiring reminders of the sheer scale of the world and the forces that have shaped it.
If there is one experience in the pharmaceutical, healthcare, and regulatory worlds that has the same potential to stun observers and shape industries, it is without a doubt an FDA AdCom. You could be the most seasoned physician, regulator, or pharmaceutical executive, but I daresay you can never sufficiently imagine or quantify the amount of expertise, time, and data required to demonstrate the many scientific and clinical angles of a drug.
Overall, bluebird bio’s recent FDA AdCom was a 15-hour extravaganza evaluating the company’s two ex-vivo gene therapies: eli-cel in cerebral adrenoleukodystrophy (CALD) and beti-cel in β-thalassemia. As the hours, presentations, and Q&A sessions progressed, I couldn’t help but think of the phrase “it takes a village.” Though it was not my first AdCom, I was still in awe of just how much brainpower must go into telling the scientific and clinical data-driven “story” of a drug’s journey through the clinic.
It's often said that no two advanced therapies are the same — and bluebird’s two gene therapies were fantastic demonstrations of this fact. However, that doesn’t mean their experience can’t be a guide for other CGT companies striving to have their own therapies evaluated by the FDA and its advisory committees.
Below, I’ve boiled down the 15 dizzyingly technical hours into three overarching takeaways for the industry. Though the FDA’s Wilson Bryan himself emphasized that clinical safety, as opposed to CMC, was the focus of discussion for the advisory committee meetings, there were a few snippets of conversation that gave us a good picture of the current CMC struggles in this nascent industry. In the following three segments, I’ll strive to showcase the higher-level takeaways that speak to the opportunities and limitations facing CGTs and their all-important CMC frameworks.
- Risk Vs. Benefit A “Living” Measurement For Commercialized CGTs
- Your Viral Vector’s Future Is In The Eye Of The Beholder
- For Cell & Gene Manufacturing, FDA Approval Is Just One Of Many Milestones