Sometimes I have answers for you, or at least hypotheses, but today all I’ve got is questions.
- Where is burosumab manufactured for use in the U.S.? I believe it’s in Japan, but I’m not at all sure and no one I’ve reached out to has been able to give me a definite answer. It matters though, because of threatened tariffs (up to 200%) on imported pharmaceuticals, and I’m sure you know that the importer (pharma company), not the exporter, pays the tariffs, and in most cases, some or all of that increase in cost gets passed along to the retail customer (patients/insurers). I can’t imagine that a pharmaceutical company would simply absorb a tripling of their costs without passing it along.
- Kyowa Kirin NA has been working on building a lab in North Carolina, which I believe will be able to manufacture burosumab, but it looks like it won’t be operational until at least 2027, more likely 2028, so I’m wondering what will happen in the interim if tariffs are imposed. Alternatively, I believe Ultragenyx already has a lab operating in Massachusetts, specifically designed for monoclonal antibodies, so I wonder if KKNA could contract with UGX to manufacture burosumab as a bridge to avoid the tariffs.
- What’s happening with the alternative treatment, via a different pathway, for which preliminary steps were taken at the FDA? Has anyone heard anything about this option that targets sclerostin?
- Is anyone moving forward with gene/cell therapy based on the proof-of-concept research in the mouse model?
- How do we reach the patients (and clinicians) who STILL don’t know there’s an effective treatment for XLH, some seven-plus years after FDA approval of burosumab? I recently read about a 30-something seeking orthopedic treatment for an XLH-related pseudofracture, and it was pretty clear that neither he nor his surgeon knew that it was a common XLH symptom, that there was a pharmacological option, or that burosumab treatment would likely render surgery unnecessary.
- How can we convince Kyowa Kirin NA to seek approval of a self-injectable option for burosumab? Especially now that a pre-filled syringe has been approved in Japan?
- Will there be any more non-profit, pure-science research into XLH, now that the NIH is fatally wounded, and policymakers aren’t interested in rare disorders?
- Has anyone looked into a shelf-stable (no freezing/refrigeration) version of burosumab? It would simplify access/shipping, and be particularly good for young adults and others whose living situation is in transition, and who may not have immediate access to a secure refrigerator.
- And finally, what’s going to happen when the 10-year followup study after burosumab was approved by the FDA ends in three years? Ideally, it should be extended for another ten years, in which case we’d have data across a whole generation, especially if the younger patients in the study are willing to sign up for the extension. Wouldn’t it be amazing to have a full 20 years of data, using a consistent protocol? Then maybe all the skeptics (mostly hide-bound clinicians who don’t like change, and insurers looking for an excuse to not pay for an expensive treatment) will stop saying, “we need to know the long-term effects before we provide this truly life-changing treatment, and instead want patients to suffer with the ineffective and harmful old treatment.”
I didn’t say they’d be cheerful questions! But those are some of the things I wonder about on the too-frequent nights when I can’t sleep. What are your XLH-related (or more general health care related) questions?
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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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