Alexion Ultra-Rare Disease Candidate Makes “Breakthrough” List
This article was originally published in The Pink Sheet Daily
Alexion’s asfotase alfa, for treatment of a life-threatening enzyme deficiency disorder, becomes the third rare disease therapy to be awarded FDA’s new breakthrough designation.
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Alexion plans to use voucher for one of its own products and might be targeting its next-generation version of blockbuster Soliris.
Companies receiving breakthrough designations seem just as unclear as investors about the value of the program.
Martin Mackay, who left AstraZeneca at the end of January after new CEO Pasqual Soriot eliminated his position as head of global R&D, is joining Alexion’s senior management team, even as the highly successful, rare disease-focused biotech seeks to put manufacturing woes behind it.