Regulatory

Sage appears set for a breakthrough FDA OK on postpartum depression drug, but who’s going to use it?

Sage can start this week with a fairly firm idea on the timeline for its initial transformation into a marketing operation, with the FDA and its outside experts appearing to bless their landmark drug brexanolone for postpartum depression. What they don’t know is who is going to buy it.

The FDA’s expert panel voted to back the drug’s risk/benefit profile by a wide margin, 17 in favor and only 1 against, with several of the votes cast with an enthusiastic endorsement for the potential here. Coming in the wake of an upbeat internal assessment at the agency, the writing appears to be on the wall for an OK by the December 19 PDUFA date.

But the same sources have also indicated that for a new drug like this, tied to episodes of unconsciousness among the small group of patients who have taken a drug designed for a very large population — something that may well have scuttled any application ahead of the liberal regulatory era we’re in now — it also appears likely that the approval will come with a requirement that patients undergo the burdensome 72-hour infusion process under 24/7 medical care. And that’s likely to severely rein in demand for the drug.

That sentiment may have weighed down the biotech’s share price. The stock was down 5% in Monday morning trading.

Paul Matteis

Stifel’s Paul Matteis was comfortable with the apparent regulatory attitude on abuse potential, leaving the door open to an approach that works for sleeping pills. But the experts balked at home infusion, wanting a set of designated facilities that can infuse the therapy over 60 hours with a 12-hour followup on safety.

Although it was clear that FDA (and of course Sage $SAGE) would have liked to be able to OK in-home infusions and minimize the burden for post-partum mothers, the agency – and the panelists – saw (loss of consciousness) risks as too salient and too unpredictable to allow for use without rigorous monitoring. It remains to be seen how specific FDA will be with the type of center in which it allows for use, but overall the discussion here was consistent with what one would’ve expected from the briefing book. Panelists asked for a variety of things like clear treatment protocols, specifics on monitoring, and a few even mentioned a black box warning for “falls” would be a smart add; FDA noted it still needs to hammer out the details of a REMS/label, but its proposal for the former is to certify healthcare facilities and have a certain list of criteria that a facility can attest to (continuous monitoring through 12-hours post infusion). On what additional data could be collected for home infusions the panel was pretty vague, hoping for a large post-marketing safety database and/or studies that could look at the efficacy of shorter duration regimens.

Add it all up, and Matteis is making a prediction of $300 million in peak sales.

The market, though, is less interested in the strings put on this approval than the understanding they can glean about the FDA’s approach to their oral followup SAGE-217. That drug is the key to unlocking the blockbuster market available here. And so far, so good, with regulators gearing up to review a drug that has — so far — not been tied to the same safety issues. 

With safety data on 140 patients, Sage is still walking on an incredibly risky tightrope as it navigates its way into the market. Once used in a bigger population, the drug is ripe for controversy if new issues arise. On the other hand, if Sage masters the change and gets green-lighted for PPD, it will be well on its way to justifying its multibillion-dollar valuation. 

The stakes are enormous.


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Molecular Templates Austin, TX
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Molecular Templates Austin, TX
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Flagship Pioneering Cambridge, MA

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