So how come XLH research never involves chocolate bars? We only get leaky buckets (see minute 1:37 of the video for a really nicely done use of the image)! Oh, but we do get cats — did you know that their purr has the same sound frequency as what’s been identified as the range that’s helpful for bone density and bone healing? So, in theory, if our bones hurt, we could cuddle a few cats and skip the doctor. (Just kidding; don’t take medical advice from me or anyone on the internet, other than the advice that if you’re hurting beyond what’s normal for you, do check with your doctor!)
On a more serious note: a reminder that pharmaceutical companies, even when they are doing good work, are still businesses with a profit motive first and foremost. They use the profits from one product (medical treatment, in this case) to enable them to develop more money-making products (other medical treatments). I don’t have any inside information on Ultragenyx, but from a recent press release, it looks like that’s what they’re doing now, essentially trading some of their future income from burosumab for current cash flow to enable them to develop similarly life-changing treatments for other rare disorders. You can see at their website (really nice graphic!) that they have quite a few products in various clinical trial phases, which means they’re either almost ready for submission of a New Drug Application for each of them or they’ve recently submitted the NDA. And that’s when things get REALLY expensive. Like three million dollars just to file each application (not counting all the salaries and other expenses that go into preparing the application and gathering the data for it)! The recent sale of some of their future income from burosumab to an investor for five hundred million dollars will come in handy for the application fees and the ongoing clinical trial work (no real idea of what that costs, but definitely multiple millions of dollars, possibly multiple hundreds of millions of dollars for each product). I only mention it in case you saw the press release (linked above) and were worried that it meant something negative about burosumab, when it totally makes sense from a business point of view.
And speaking of business points of view … when is someone going to reach out to patients about the change in responsibility for the management of burosumab in North America from Ultragenyx to Kyowa Kirin? If you don’t already know, Kyowa Kirin (the original company in Japan, not the newer company in the U.S.) was the initial developer of burosumab (based on the discovery of FGF23 by Dr. Econs, famous for his work with chronic hypophosphatemias). That initial product was then licensed to Ultragenyx to get marketing approvals and then manage the product in various countries, including North America, for a set number of years. That timeframe expires at the end of this year, and then, I believe, Kyowa Kirin will take over responsibility for burosumab. Before you ask, I don’t know exactly what that means for patients. Ideally, the transition will be seamless, but I’d really like some assurance of what’s happening to make that so. I’m cautiously optimistic, because I’ve seen the great patient-focused work another iteration of Kyowa Kirin (there are separate but sort of related entities in various parts of the world) is doing in Europe. I’d still like some sort of formal explanation of what’s going on!
Here’s a new document from Australia’s Therapeutic Goods Administration about burosumab that I don’t really understand the purpose of, but it’s got some good information, including a table that shows the status of the approval process in various countries. And you’ll note that it was prepared in cooperation with Kyowa Kirin Australia, another regional iteration of Kyowa Kirin.
Do you like to read science fiction or have a kid who does? Check out the Vorkosigan saga by Lois McMaster Bujold. The first book is about the mother of Miles Vorkosigan, but most of the rest are centered on the son who has a rare bone disorder that any XLHer can empathize with. Miles is an extraordinary character, who doesn’t let a few broken bones (or even a whole-body cast) keep him from doing what he wants to do. His condition is a little more like Osteogenesis Imperfecta, with brittle bones, than XLH with soft bones, but there’s a lot in his experience that XLHers would empathize with.
And if you have a younger kid with XLH, check out Max and Milo, by a fellow XLHer and mother-to-XLHers.
And if you want to see what else I write (with apologies for this little bit of self-promo), check out A Dose of Death for free (at Amazon only, digital format only, through September 25th), featuring an amateur sleuth facing ultra-early retirement (in her forties) due to a health condition (lupus, not XLH), and getting really cranky as everyone underestimates her due to her chronic illness. I think you’ll appreciate some of her struggles!
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Please note that the author is a well-read patient, not a doctor, and is not offering medical or legal advice.
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