Alexion bags another FDA 'breakthrough' for rare-disease drug

Alexion Pharmaceuticals ($ALXN) has racked up its second breakthrough therapy designation from the FDA, securing a faster track for an early-phase enzyme-replacement treatment targeting an ultra-rare metabolic disorder.

The agency has smiled on ALXN1101, which Alexion picked up from Orphatec for $3 million plus milestones, putting the rare-disease treatment alongside the company's Phase III asfotase alfa, which joined the list of drugs tabbed for expedited approval in May.

ALXN1101 is designed to treat molybdenum cofactor deficiency (MoCD) Type A, a fatal disorder that leaves newborns with catastrophic brain damage in the first weeks of life. The rare disease results from a lack of the enzyme cyclic pyranopterin monophosphate (cPMP), and Alexion's drug provides a synthetic replacement that has shown promise in early investigation, the company said.

"In designating cPMP as a breakthrough therapy, the FDA recognizes the life-threatening nature of MoCD Type A, the positive early clinical results associated with cPMP and the lack of any effective treatment options for infants born with this devastating disease," Alexion R&D Chief Martin Mackay said in a statement.

Now, with the FDA's recognition, the company figures it's in line for a speedy trip through development, but with the industry still in something of a feeling-out period with the agency's breakthrough label, it's unclear just what kind of difference the designation will make. Touting the program earlier this year, Center for Drug Evaluation and Research Director Janet Woodcock said drugs could well win approval based on Phase I results, provided the data were compelling and disease target dire, but that scenario has yet to become a reality.

For Alexion, now that all those Roche ($RHHBY) buyout rumors have subsided, the prospect of near-term approvals for two rare-disease treatments gives it a chance to build on its marketing successes in the field. Soliris, the company's lone approved drug, is the most expensive in the world, and Alexion is expecting to book about $1.5 billion in revenue this year.

- read the statement

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