Alexion's Soliris didn't ace its myasthenia gravis trial, but it might still win big sales

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Alexion Pharmaceuticals ($ALXN) wants to take its ultra-orphan drug Soliris into yet another disease: myasthenia gravis, a rare neuromuscular disorder. After all, at a list price of $700,000, every new patient added to the drug’s target market adds new punch to the biotech’s top line.

Thing is, the drug didn’t hit its primary goal in a key myasthenia gravis trial unveiled this week, and that performance could put a new FDA approval in jeopardy.

But if doctors continue to use the med off-label, that FDA stamp of approval might not matter as much as common sense might say.

Doctors may do just that, analysts said this week, citing interviews with leading specialists who treat the disease. Apparently, the physicians still see Soliris’ performance in the REGAIN trial as “positive,” and some believe the FDA will grant the new indication despite the primary endpoint miss.

The drug did narrowly miss that endpoint--improvement in a common clinical measurement of the disease--and it hit three secondary goals. Patients’ scores on that clinical tool did show improvement that was “clinically meaningful,” one doc told analysts at SunTrust.

Plus, the population Soliris is targeting is hard-to-treat patients, who have “substantial morbidity and disability,” and need expensive medical care, one top specialist told Leerink Partners.

“[T]he treatment effects were highly significant clinically, and even the treatment cost could be justified based on the high cost of care for these severe MG patients,” Leerink analyst Geoffrey Porges wrote in a Tuesday note after doctor interviews.

To those specialists--and others who’ve dug into the nitty-gritty statistical analysis in REGAIN--the “totality” of the data still justify its use, and given the severe symptoms these hard-to-treat patients suffer, they and their doctors are likely to try the med.

“The overwhelming preponderance of evidence suggests that the drug is active, and safe, in this disease,” Porges said, predicting that the FDA will eventually give the new use its blessing. SunTrust’s analysts peg the possibility at 65%.

The question is whether payers will be willing to shell out for the ultra-expensive drug without an FDA go-ahead. Given the high cost of care analysts highlighted, they might see it as a money-saving proposition, even at $700,000 sticker price--and they might be able to squeeze bigger discounts out of Alexion for the off-label use. Then again, Alexion hasn’t had much success with skeptical payers lately; the cost-effectiveness gatekeepers in Canada recently stiff-armed the drug after pricing negotiations failed, and England’s NICE demanded more justification for the high price on the company’s new rare disease med Kanuma.

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