So yes, pheochromocytomas and paragangliomas are quite difficult to study. And there are, I think, two main reasons for that. Number one, they are quite rare in clinical practice. If you look at the statistics in the U.S., we may see 1,000 to 2,000 new cases of pheochromocytoma or paraganglioma every year. One of those, 200 to 300 cases are metastatic. So it’s what we call, by definition, an orphan disease...
So yes, pheochromocytomas and paragangliomas are quite difficult to study. And there are, I think, two main reasons for that. Number one, they are quite rare in clinical practice. If you look at the statistics in the U.S., we may see 1,000 to 2,000 new cases of pheochromocytoma or paraganglioma every year. One of those, 200 to 300 cases are metastatic. So it’s what we call, by definition, an orphan disease. And that has made it quite difficult to make progress regarding systemic therapies for these patients. So that’s one of the important aspects. The second one is how heterogeneous the disease is. It’s a disease that is difficult to predict. There are some patients in whom the disease is quite stable and doesn’t bother them despite being metastatic, and those patients may just benefit from observation. There are some patients in whom the disease is quite aggressive, rapidly progressive, and they may die in a matter of months. The majority of patients exhibit progression over time, and they will require some type of systemic therapy at some point. Just because the hormonal activity of the tumors, the large tumor burden, and the disease progression, yes, may kill many of these individuals and may lead to an impressive morbidity rate. With medications such as Belzutifan, at least in a substantial number of patients, we’ve been able to show that we are able to improve the tumor burden, the hormonal activity, and we are able to slow down or stabilize the disease preventing additional progression. So the medication is helping a lot. When you look at the history of pheochromocytomas and paragangliomas, there was another FDA-approved therapy. That one was high-specific activity, MIBG. Unfortunately, it has been discontinued from clinical practice. It was discontinued last year, in part because of how expensive the production was, in part because of the lack of demand and because of that for some time we were not able to get any treatments but now we have another one and hopefully this is a medication that becomes a standard of care in many other countries around the world.
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