- An Advisory Committee to the Food and Drug Administration has recommended the approval of a rare disease drug from Ionis Pharmaceuticals and Akcea Therapeutics, the companies said Thursday.
- In a 12-8 vote, the committee determined the safety and efficacy data for volanesorsen would be sufficient to clear it for patients with familial chylomicronemia syndrome (FCS). The FDA isn't bound to follow advisory committee recommendations, but usually does.
- Ionis and Akcea intend to market their drug under the brand name Waylivra. They expect to hear an approval decision from the FDA by Aug. 30.
FCS is an inherited disease that inhibits the metabolism of certain fats, in turn causing a build up of triglycerides in blood plasma. Late-stage clinical testing showed patients treated with volanesorsen on average experienced a 77% reduction in plasma triglyceride levels from baseline.
But the drug also elicited some safety concerns, namely reduced blood platelet counts. The reductions were a main concern to FDA representatives, who argued a risk evaluation and mitigation strategy, or REMS, be required for volanesorsen if it gains approval.
While the advisory committee's vote increases the likelihood of that outcome, regulators don't seem entirely sold on volanesorsen's efficacy and safety profile. FDA representatives, for instance, zeroed in on clinical analyses that showed Ionis and Akcea's drug offered no significant benefit to common symptoms of FCS such as abdominal pain or pancreatitis.
In a May 10 investor note, EvercoreISI analyst Joshua Schimmer acknowledged it's still unclear whether "the FDA can get comfortable with an approvable framework for the drug." Given the safety signals, the agency may approve volanesorsen only for ultra-high risk patients, or ones with particularly high triglyceride levels, Schimmer wrote.
Such caveats threaten the sales outlook for volanesorsen, so much so that EvercoreISI has taken the drug out of its model for Ionis. The investment bank earlier this week downgraded the biotech's shares from "Outperform" to "In-Line." The committee's vote wasn't enough to reverse the tides.
It was "technically a positive panel, but one which does not substantially increase our enthusiasm for the commercial prospects, assuming approval," Schimmer wrote.
Akcea and Ionis, as well as their investors, were more optimistic. Both took double-digit stock hits earlier this week when briefing documents indicated safety would be a central focus at the advisory committee meeting. But Akcea, which is majority-owned by Ionis, saw shares more than rebound Friday morning to trade at $23.54 apiece.
Ionis stock had a more modest recovery, with shares rising by more than 5% to near $45 per share.