If you’ve followed my blog for the last year or so, you’ve seen that I’ve been repeatedly discussing the value of generic medicines. The brand we’ve long known as Kalydeco will soon be forced to compete with a generic equivalent, and that’s a good thing! Generic medicines, typically, provide seemingly unlimited value to patients and...
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With news of the emerging biotech company, Sionna, coming out of stealth yesterday, there is, once again, a reinvigorated hope for more transformational cystic fibrosis medicines on the horizon. In case you missed it, Sionna is attempting to correct CFTR with a small molecule drug like Vertex’s class of modulators but is targeting a different...
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Gene therapies are all the rage these days in the world of emerging biotech. It’s not hard to understand why. It has never been cheaper (on a giant scale) to understand human genetics and the human genome. We’re also living at a point in time where technologies to deliver whole genes or “correct” existing genes...
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If Mark Cuban’s Cost Plus Drug Company (yes, that’s literally the name) can be sustained, it might be the welcome relief that can drive generic drug prices down for consumers and reassert the kind of balance Hatch-Waxman had intended to establish when the law was passed in the 1980’s. The new venture has been all...
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Salt in My Soul, the documentary version, is something special. If you’ve followed the blog for a long time, you know I loved the book. In fact, Darcy and I had a passage from Salt in My Soul read aloud at our wedding: My life is a miracle. Life in general is a miracle. Our...
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The Cystic Fibrosis Foundation has started off the new year with a handful of new investments that suggest the cystic fibrosis market is accelerated, and not decelerating as some had feared. Not long after Trikafta was approved by the FDA, the world rightly celebrated such an important milestone in the history of biomedical innovation. Cystic...
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Last week we celebrated news out of the North American Cystic Fibrosis Conference that median age of survival (remember, the age beyond which we expect 50% of people with CF born today to live, under the assumption that recent age-specific mortality rates will hold for the rest of their lives) climbed to 50 years old,...
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