Purple Canaries

Joyce Gould with Jill Gould

National Porphyria Awareness Week 2020 – 4/22

Continued – 4/22

It took nearly three decades for APF to make its next big financially-driven motive-move—welcoming Alnylam Pharmaceuticals into the alliance (~2013). This was no doubt predicated on knowing that more governmental incentives were coming down the pike, namely Obama’s 2015 Precision Medicine Initiative and the 21st Century Cures Act (2016). By then Mount Sinai geneticist/APF SAB member Desnick had abandoned researching and promoting his AIP molecular diagnostic technique (essentially a pre-cursor to genetic testing) in anticipation of genetic testing becoming the leading edge of acute porphyria diagnostics (aka “precision medicine). With the Orphan Drug Act (ODA) of 1983 as its backdrop, the clear aim of the APF/Anlylam partnership was to develop a “game changing” orphan drug  treatment for AIP (actually, as it turned out, for all the rare acute porphyrias) to substantially up the ante for increasing APF et al’s wealth and power. Givlaari® earned marketing approval in November 2019, only four months after a new drug application had been submitted to the FDA. Right from the get go, Alnylam took the wheel in managing worldwide public awareness of the acute porphyrias. By May 2013, the four acute porphyrias went from having been individually identified as rare to the bundling of the four acute types (AIP, ADP, HCP, VP) together under the label of AHP (acute hepatic porphyria) with the entire disease group heralded as “ultra rare.” With very few exceptions the aforementioned audience(s) bought it hook, line and sinker. In demonstrating BLT-flavored APF’s bended knee to Alnylam’s lead, a Purple Light Blog post read, “Porphyria is just one of those ultra rare diseases in the form of Acute Hepatic Porphyria or Cutaneous type of Porphyria.” Henry Waxman, primary sponsor of the ODA, had hit the nail on the head when in 2013 he said, “The [pharmaceutical] industry has taken advantage of the incentives to charge excessive profits and to reap windfalls far in excess of their investments in the drug.” Indeed.


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