We are closing in on the end of another week in the biotechnology space just as was the case last week, we’ve seen plenty of movement in the sector on the back of a number of fresh releases. Here is a look at the most pertinent and a description of what it means for the companies in question.
Akcea Therapeutics, Inc. (NASDAQ:AKCA) and Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) are two of the biggest movers at the end of this week, with the action rooted in the release of an outcome report from an advisory panel meeting.
Specifically, these two companies are trying to bring a drug to market called WAYLIVRA (volanesorsen) in a target indication of what’s called familial chylomicronemia syndrome (FCS). For anybody not familiar with this condition, it is a rare genetic disease characterized by the build-up of chylomicrons (chylomicronemia), the largest lipoprotein particle, which are responsible for transporting dietary fat and cholesterol. Typically, the enzyme lipoprotein lipase (LPL) breaks down chylomicrons in the blood but, in people living with FCS, LPL does not function properly. This inability to break down chylomicrons1-3 leads to an increase in triglyceride levels and characterizes the condition.
So, right now, treatment options for patients suffering from FCS are extremely limited and the condition can be debilitating and potentially life-threatening if left to progress untreated.
With volanesorsen, then, Akcea and Ionis are attempting to remedy the fact that these patients have very little in the way of options and it’s this attempt to which the latest development to hit press relates.
Specifically, and as mentioned above, the drug was recently the focus of a committee meeting set up to allow for experts to discourse and subsequently vote upon the approval ability (or otherwise) of the program in question.
These sorts of meetings are usually set up by the FDA in the US, with the latter then using the outcome (as determined by the above-mentioned vote) to inform its decision on whether or not to approve the drug in its current iteration. It’s important to note that the FDA isn’t obliged to follow the recommendation (i.e., if a panel votes against approval and the majority, the FDA doesn’t automatically need to turn the drug down, it could still approve it) but more often than not, if a panel votes in favor of approval then the FDA will fall in line with this recommendation.
So what happened in this instance?
Well, it’s pretty good news for the companies involved and their respective shareholders.
On the back of the meeting, panel members voted in favor of recommending approval, with the numbers showing a 12 to 8 majority in favor of said recommendation. As such, the implication here is that more experts in this arena felt that the drug had a favorable risk-benefit profile and that, given the rarity of this disease and its associated lack of available treatment options, then approval would benefit the US population.
And as might be expected, both companies have picked up a boost in share price on the back of the news.
And what happens next?
Now, the focus turns to the PDUFA date for the drug. This is the date that the FDA put in place when it first accepted the NDA for review (subsequent to submission) and it is the date by which the agency will make a decision as to whether or not it wants to approve the asset, with the recent recommendation being used as guidance.
In this instance, the date is set for August 30, 2018, and we will likely see some degree of speculative loading moving towards this event and a potential catalyst for both companies.
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