Good morning, everyone, and welcome to another working week. We hope the weekend respite was relaxing and invigorating, because the usual routine of online meetings and deadlines has predictably returned. But what can you do? The world, such as it is, continues to spin. So time to give it a nudge in a better direction with a cup or three of stimulation. Today, we are reaching for hazelnut creme. As always, you are invited to join us. Meanwhile, here are a few items of interest to help you along. We hope you have a meaningful and productive day. And of course, do keep in touch. We appreciate feedback and tips. …
The U.S. Food and Drug Administration’s historic approval of the first gene therapies for sickle cell disease sparked discussion, debate, and, above all, measured optimism at this year’s meeting of the American Society of Hematology, STAT says. Doctors and researchers noted both therapies marked a landmark shift in the treatment of sickle cell, a disease that has long been ignored and underfunded. But they also questioned whether these cutting-edge treatments will reach the patients who need them most, citing issues ranging from their cost to the limited number of treatment centers, as well as the challenge of educating both patients and health care providers about the medicines.
The U.S. regulatory approval of Bluebird Bio’s gene therapy for sickle cell disease should have been a momentum-swinging achievement for the long-struggling biotech. Instead, the company mispriced its new drug and fumbled a pivotal financial lifeline, STAT explains. The consequences of these strategic blunders — arguably, self-inflicted — could imperil Bluebird’s independence, perhaps even its survival. Bluebird priced its therapy at $3.1 million, much higher than the competing Vertex Pharmaceuticals treatment. Its prescribing label carries a “black box” safety warning to monitor for cancer risk. And it failed to win a regulatory voucher to help with the commercial launch.
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