FDA enforcement discretion to allow unregistered generic Daraprim to be imported and sold at lower prices

In 2011, the FDA told the NIH it could use enforcement discretion to permit importation and sale of unregistered drugs

In 2011, in the context of a shortage of Fabrazyme, the FDA told the NIH if they could "in theory" allow Shire to import and sell Replagal, a drug approved for sale in Europe, but not in the United States, as an unregistered biologic substitute. The remedy explored for the Fabrazyme shortage may be the best way to address a different problem, the price gouging associated with the drug Daraprim (generic name: pyrimethamine).

Fabrazyme is the Genzyme/Sanofi brand for an enzyme replacement therapy to treat Fabry disease, an inherited disorder caused by deficiency of the alpha-galactosidase A enzyme. Ultimately, Sanofi, Shire and the Icahn School of Medicine at Mount Sinai entered into a series of agreements that left the United States with only one supplier for an enzyme replacement therapy to treat Fabry disease (http://keionline.org/node/2055).

Daraprime has been on the market in the United States since 1953, and there are no patents for the drug listed in the FDA Orange Book. Outside the United States, there are plenty of generic versions of this drug with prices as low as 2 cents per pill. Turing Pharmaceuticals has increased the price in the United States to $750 per pill, an aggressive action that has attracted considerable public interest and outrage.

The notion that the FDA could use its enforcement discretion to allow the importation and sale of an unregistered drug is a possible and appealing way for the Obama Administration to address Turing's aggressive price gouging. The generic alternatives sold outside of the United States include products that have received marketing approval from high standard regulatory agencies, so there is no reason to think they present a health risk.

Below is the August 5, 2011 email sent by Kathy Hudson, the Deputy Director for Science, Outreach, and Policy at the National Institutes of Health (NIH), to Francis Collins, the Director of the NIH. (Note also that Hudson recommended the NIH Director grant the Fabrazyme March-In request).

From: Hudson, Kathy (NIH/OD) [E]
Sent: Friday, August 05, 2011 2:31 PM
To: Collins, Francis (NIH/OD) [E]
Cc: Tabak, Lawrence (NIH/OD) [E]; McGarey, Barbara (NIH/OD) [E]; Devaney, Stephanie (NIH/OD) [E];
Hudson, Kathy (NIH/OD) [E]
Subject: Fabry's - it is time to act

Hi,
Last week we spoke to FDA who said that “in theory” they could use enforcement discretion to permit Shire to market replagal in the US.

Yesterday Genzyme reported a new problem with fabryzyme supply that some are reporting as NO enzyme for month of august (gulp…) http://www.pharmalot.com/2011/08/genzymepatients-angry-over-fabrazyme-supplies/ and pasted below

I talked to Shire today about whether, in a hypothetical scenario, they would be able to supply replagal to cover drug shortage in the US. I was told that they would be able to provide HRT to hundreds of patients immediately growing to full supply to end shortage in 4 months though she was doing back of envelope and would need to get better data if in fact we had a magic wand to wave.

I would like to discuss this with you but I would propose that we:

1. Ask FDA to give replagal temporary okay to sell in US. We might ask KGS to back us….
2. Set up call with mt sinai, genzyme, and sanofi and say that we are prepared to march in unless they give Shire a license for US and Europe at reasonable costs and that the deal be done immediately.

Patients will only be helped in the short term if (1) happens.
Patients will only be helped in the long term if both 1 and 2 happen.
I think we should act right away… This is heart breaking.

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