Last week Alnylam reported positive news on Phase 3 outcomes for their RNA interference (RNAi) therapy to treat patients with a rare genetic cause of amyloidosis with polyneuropathy (see here). I tweeted the following:
The 20-year journey from scientific discovery to positive Phase 3 clinical trial data got me thinking about other novel therapeutic modalities. Was twenty years a long time or typical for an innovative therapeutic modality? Where are other promising modalities on their journey to regulatory approval? Is the biopharmaceutical industry on the cusp of a series of innovative modalities that could change the therapeutic landscape for patients? How will these new modalities improve our ability to test therapeutic hypotheses?
[Disclaimer: I am an employee of Celgene. The views expressed here are my own.]
To explore these questions, I decided to review different novel therapeutic modalities, which I am defining as those other than small molecules, protein therapeutics (e.g., insulin) and traditional vaccines. This decision was practical, as the amount of literature in these modalities is expansive.
For each new modality, I asked whether a drug has been approved by either a European or US regulatory agency (EMA and FDA, respectively). If a drug has been approved, I reviewed the time from seminal scientific discovery (which sometimes is clear, sometimes is not) to the approved therapy.…