Purple Canaries

Joyce Gould with Jill Gould

National Porphyria Awareness Week – 4/24


The month before givosiran received FDA approval, an investor analyst questioned AHP prevalence numbers, apparently seeking reassurance that market conditions would support financial expectations now and in the future as the potential for 5,000 patients had been mentioned before.  Alnylam’s president responded, “as we get really granular on the patients that have active disease and are diagnosed, there are a total — we believe there are a total of 3,000 in the US and Europe, obviously a larger number in the rest of world. But 3,000 in the US and Europe, of which 1,000 of those 3,000 are those recurrent attack patients that have four attacks or more and then 2,000 of those 3,000 are the less recurrent patients…when we talk about actual diagnosed patients, we’ve always said that 5,000 number.”

Obviously, the numbers stated from all parties since 2013 are ridiculously beneath the <200,000 ODA requirement for orphan disease patient population. Looking back on the slide citing 2,543 AHP patients in APF’s patient registry, how in hell, could there have been only 3,000 patients in the US and Europe four years later? Knowing that there are tens, hundreds and maybe thousands more expert-rejected patients in the US and Europe—and the entire world, these nonsensical numbers are laughable. Yet in spite of pabulum behind it and a drug safety profile [that] “left much to be desired”, Givlaari, the “Drug for Ultra Rare Disease Comes with Ultra High Price Tag” received FDA approval in November 2019. It’s no wonder that APF toes the line. Desiree Lyon’s ardent protectionism in keeping acute porphyria rare was revealed during the APF sponsored March 1, 2017 “Patient Focused Drug Development Meeting with the FDA—Acute Porphyrias.” On the video she is heard interrupting a querying patient trying to make her point, “there are a lot people with porphyria now…and it is still rare because there’s no knowledge….” Lyon jumped in, “No, it’s rare because of the number of patients. It’s not rare because of the knowledge, it’s rare because of the number of patients. The number of patients.”

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