STAT Plus: Pharmalittle: Bluebird Bio prices gene therapy at $1.8 million; lawmakers urge feds to step up generic probe
And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda is rather modest. We plan to escort Mrs. Pharmalot to a Rent Party, hang with one of our short people, promenade the official mascot, and catch up on some reading. And what about you? This is a lovely time to enjoy the great outdoors — beaches, lakes, and mountains are beckoning — or think ahead to a summer getaway. Or if you feel like taking a risk, you could place a bet on which countries might offer you-know-who some political dirt. Well, whatever you do, have a grand time. But be safe. And if Dad is around, remember to say hello. Enjoy, and see you soon. …
Bluebird Bio (BLUE) will be selling the world’s second-most expensive drug, now that it has priced its Zynteglo, a gene therapy for a rare blood disorder, at $1.8 million, STAT reports. Only Zolgensma, the Novartis (NVS) gene therapy for spinal muscular atrophy, is priced higher, at $2.1 million. Bluebird is offering to put as much as 80% of Zynteglo revenue at risk — meaning the biotech would not receive payment — if the medicine fails to benefit patients.
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