Last year was unquestionably an excellent year from an FDA NCE approval perspective. A recent article in Nature Reviews Drug Discovery has the full list. An interactive table (complete with chemical structures) is also available here.
So what stands out from all of these approvals?
HIV Approvals – Remember all of the attention directed towards AIDS during the 80s and 90s? While we have made great strides in managing the disease, the disease has not gone away.
New infections have been declining since the mid-1990s, and patients are living much longer with the disease. Yet we still see three approvals in 2018 for HIV.
This is terrific, as there is still a need to reduce pill burden, push life expectancies even further, and have options for those patients who experience drug resistance.
Cannabidiol – Many said cannabidiol (“CBD”) would never be approved for anything. Yet, here we are, with an approved formulation of plant-extracted CBD for the treatment of several rare forms of epilepsy.
It’s too early to accurately forecast what the approval of CBD will mean for the broader cannabis markets. But could we see this product used off label for pain and inflammation? Multiple sclerosis? Others?
Perhaps not, given our pricing and reimbursement environment. However, there is a lot of pharmacology to be explored here. And we hope this is the first of many further approvals in the cannabis space.
Blockbusters – A separate analysis projects that peak year sales (“PYS”)for these newly approved products will be “only” $720 million each on average, resulting in combined peak sales of $45 billion for this cohort of new products. Peak sales estimates are notoriously tricky to get right, so we are not convinced these figures are that meaningful.
However, if it is true that average PYS for newly approved products are declining, then it could be that we are developing more products for increasingly well-defined, narrower indications. This is consistent with the observations made by BIO and others, that the industry is moving away from indications that require large clinical programs and large sales efforts.
Not One, Not Two, But… – Three new products for the same indication leveraging the same mechanism of action were approved in 2018. Three CGRP antagonists for the treatment of migraine were approved. Interestingly, all three are monoclonal antibodies.
Have we completely given up on small molecule CGRP antagonists? Perhaps, as oral bioavailability and other issues have plagued oral CGRP antagonists.
River Blindness – A drug called moxidectin was approved for the treatment of River Blindness. What’s interesting about this approval? It was developed and approved by Medicines Development for Global Health, an Australia-based not-for-profit which focuses on prevalent diseases in low- and middle-income countries.
Looking at their management team, there is a lot of pharma experience there. This is not a celebrity-driven operation. Rather, it’s a real pharmaceutical company leveraging their non-profit status to raise capital and develop products.
Can other not-for-profits jump into the drug development arena as well? Not just sponsoring research, but actually performing research internally (or out-sourced)?
With all of the negativity surrounding our industry lately, tables like these are a good reminder of what our industry can do. Aside from the many novel cancer therapies approved last year, the list also has new therapies for the treatment of HIV, cystic fibrosis, opioid withdrawal, and severe infections.
As we’ve said many times, our industry has issues. We will continue to be the target of politicians and journalists attacking us for our alleged greed at the expense of patients.
However, these results show that we should be proud of what we do.