Update: Patents Kill: Compulsory Licenses and Genzyme’s Life Saving Drug

In my post Patents Kill: Compulsory Licenses and Genzyme’s Life Saving Drug, I noted that, due to the monopoly granted by patent (yes, it is a monopoly), people are literally dying because the drug Fabrazyme is in short supply and the sole, monopolistic manufacturer, Genzyme, can’t make enough quickly enough–and no one else is permitted to make it due to the patent.¹

In response to my blogging about this, I received an email today from (ironically) a patent attorney (appended below), C. Allen Black, Jr. Yes, I know he’s a patent attorney–but nonetheless, he is heroically representing pro bono two victims of the murderous FDA-patent regulatory system caused drug shortage. As noted in the press release issued by Black on behalf of his clients:

Mr. Joseph M. Carik was diagnosed with Fabry disease in 2005, before the shortage so he receives only a 30% dose.  Ms. Britton was diagnosed in 2010 after the shortage so she is not allowed access to the drug.  As a result of rationing, all petitioners have had their symptoms return, including pain and burning in their extremities (neuropathy); decreased kidney function (proteinuria), severe gastrointestinal symptoms, and cardiac problems.

This is an outrage–a clear example of the destruction to human life caused by the state–in particular, its patent and FDA systems. As noted above, there is a shortage because of the patent, but combined with the FDA. I frankly do not understand why Genzyme itself has not licensed its patent to others, at least for the time being, if only for humanitarian reasons. But even if they don’t the FDA/fedgov ought to impose a compulsory license, which it has the statutory power to do; and/or the FDA ought to approve the “alternative drug, Replagal® manufactured by Shire Pharmaceuticals.” This mess is the result of the intersection of state regulatory systems. I’ve noted this before in my posts State Antitrust (anti-monopoly) law versus state IP (pro-monopoly) law; When Antitrust and Patents Collide (Rambus v. FTC); The Schizo Feds: Patent Monopolies and the FTC; The Schizophrenic State; Intel v. AMD: More patent and antitrust waste; Are Patents “Monopolies”?; Patents, Prescription Drugs, and Price Controls. The FDA imposes costs and delays, and then the state and IP proponents use this as an excuse to argue for a patent system: the patent monopoly price is needed to “make up” to the pharmaceutical companies the damage they suffer from the FDA system (which is not really always a damage, since it works in some ways like a patent since the FDA might approve your drug and not those of competitors, giving you a patent-like monopoly; and also because the relatively high cost FDA compliance imposes on smaller companies–for examples of how large corporations often lobby for and benefit from (relatively speaking) antitrust and other regulations, see State Antitrust (anti-monopoly) law versus state IP (pro-monopoly) law).

In this case, as explained by Black’s email, there is a shortage because

62% of Fabrazyme (Genzyme Corp) is sent overseas even though overseas patients have access to the alternative treatment, Replagal by Shire Pharmaceuticals. Replagal is not approved for use in the US and Shire has withdrawn its FDA application for approval, so no alternative treatment exists for Americans …. The result is the tragically ironic situation that there is no drug shortage overseas, but, here in the US, no new patients receive treatment and pre-existing patients are still rationed to less than half the recommended dose. There have already been three deaths reported in the US under the rationing, but none reported overseas. Even more bizarre, is that the FDA is importing Replagal to replace the Fabrazyme that was shipped overseas in the first place. However to receive treatment the patients must show “critical medical need” on an individual basis.

The FDA-patent victims here tried to get another manufacturer into the market but the NIH said “no.” As Black told me, “The FDA is our last resort because they are the only administrative body that can allocate drug during a crisis.” So now, these poor, sick people, who are denied access to a necessary drug because of the state’s murderous regulations, have to petition and beg the FDA to use its emergency decree powers to help ameliorate a crisis that it (and the fedgov) caused in the first place. Their plight reminds of those poor, sick people who need medical marijuana but have to skulk in the shadows, live in fear, or beg their master-the-state for permission to ease their own misery.

Here are Black’s email to me and press release. Please consider signing the Citizen Petition to help these people out: it’s available at http://www.patentlawyersite.com/files/Download/Web%20CITIZEN%20PETITION%20TO%20ALLOCATE%20AGALSIDASE%20BETA.doc.

Black has more information on his clients and this situation at his page Fabrazyme and March-In Rights.

Black’s email:

Dear Stephan,

Despite the disappointing response by the NIH to the Fabrazyme drug shortage last month, we learned disturbing news from the opinion. 62% of Fabrazyme (Genzyme Corp) is sent overseas even though overseas patients have access to the alternative treatment, Replagal by Shire Pharmaceuticals. Replagal is not approved for use in the US and Shire has withdrawn its FDA application for approval, so no alternative treatment exists for Americans.

The result is the tragically ironic situation that there is no drug shortage overseas, but, here in the US, no new patients receive treatment and pre-existing patients are still rationed to less than half the recommended dose. There have already been three deaths reported in the US under the rationing, but none reported overseas. Even more bizarre, is that the FDA is importing Replagal to replace the Fabrazyme that was shipped overseas in the first place. However to receive treatment the patients must show “critical medical need” on an individual basis.

Mr. Joseph Carik who previously petitioned the NIH and Ms Amber Britton, who receives no treatment whatsoever, have filed a citizen petition with the FDA in this case to distribute drug first to US patients under the FDA consent decree with Genzyme corporation. Alternatively, the petitioners request that their case for medical need be heard before the drug is shipped overseas. The petitioners believe (as do I) that if drug was allocated to US citizens first, then there would be enough to end the rationing in the US.

I have attached the news release which links to the petition. Thank you for your interest in this story.

Best regards,
Allen

C. Allen Black, Ph.D., J.D.
1579 Montgomery Rd.
Allison Park, PA 15101
412-908-3268

[email protected]

Fabrazyme families press release:

Genzyme Fabrazyme® – FDA asked to allocate full doses of Fabrazyme® to treat Fabry Disease Patients in the U.S. prior to exporting it overseas

Washington, DC – January 19, 2011, — Today victims of the genetic illness Fabry Disease, (Joseph M. Carik of North Las Vegas, Nevada, and Amber Britton of Kirkland Washington) petitioned to allocate full doses of Fabrazyme® to U.S. Fabry patients.  Fabrazyme® is manufactured by Genzyme Corporation and it is the only approved FDA enzyme replacement treatment for Fabry disease, a relatively rare genetic disease.  Currently, no newly diagnosed patients such as Amber Britton are eligible for treatment and pre-existing patients such as Mr. Carik are rationed to less than half of the recommended dosage.  As a result patients’ symptoms have returned and patients are increasingly likely to die of the disease.  The U.S. situation is dire with as many as three deaths of Fabry patients reported during the drug shortage.

Genzyme, which produces Fabrazyme® under an exclusive license from Mt. Sinai Medical Center, has been unable to produce enough drug to treat the US Fabry disease market since mid-2009 due to various manufacturing errors.  As a result, Genzyme entered into a consent decree with the FDA in which Genzyme agreed to a fine of $175 million dollars.

However, 62% of Fabrazyme® being manufactured in the U.S. is allocated to patients overseas, even though such patients have access to the alternative drug, Replagal® manufactured by Shire Pharmaceuticals.  Since April of 2010, the European Medicines Agency has recommended that patients either receive a full dose of Fabrazyme® or be switched to Replagal®.  As a result there is no drug shortage in Europe.

In the U.S., Replagal® is not an FDA approved drug, and Shire has withdrawn its application for FDA approval, so no alternatives exist.  The U.S. is the only country in the world where Fabry patients receive no treatment for their disease, even though the discovery and patents were obtained as a result of public funding by the National Institutes of Health.

The petition filed with the FDA requests that Fabrazyme® be allocated first to U.S. patients and, if it is not, that the patients be allowed to present their case for medical need before it the drug is sent overseas.  The FDA has allowed some Fabry patients to import Replagal® from Europe for emergency medical use in the U.S.  However, patients must petition the FDA and then show a critical medical need.

The FDA has the authority to regulate distribution of drugs during a shortage under its consent decree with Genzyme®.  It has previously exercised implementing an allocation program for repository corticotropin injection (Questcor Pharmaceuticals),  caspofungin acetate (Cancidas, Merck) and beta methasone sodium phosphate (Celestone Soluspan, Schering).

A copy of the Citizen Petition is available on the web at http://www.patentlawyersite.com/files/Download/Web%20CITIZEN%20PETITION%20TO%20ALLOCATE%20AGALSIDASE%20BETA.doc

Mr. Carik previously petitioned HHS to allow manufacturers to produce Fabrazyme® under a Bayh-Dole march-in license.  Ms. Britton formally supported the petition.  However, the request was denied last month by the NIH stating that FDA regulations prevented another manufacturer from making the drug in a timely manner.  Specifically, the NIH ruled that despite the critical health need, the FDA regulations would require manufacturers more than three years to obtain FDA approval.  Thus, granting march-in rights to manufacturers would be futile.

Mr. Joseph M. Carik was diagnosed with Fabry disease in 2005, before the shortage so he receives only a 30% dose.  Ms. Britton was diagnosed in 2010 after the shortage so she is not allowed access to the drug.  As a result of rationing, all petitioners have had their symptoms return, including pain and burning in their extremities (neuropathy); decreased kidney function (proteinuria), severe gastrointestinal symptoms, and cardiac problems.

Fabry disease is a rare disorder with an estimated prevalence in the general population of 1 in 117,000 people.  Those with the disease are unable to metabolize fats properly leading to numerous symptoms, the most serious of which are renal failure and degenerative heart disease.  Most patients did not live much beyond 50 prior to the development of enzyme replacement therapy such as Fabrazyme®.

The petitioners are represented pro bono in the matter by C. Allen Black, Ph.D. who is a licensed patent attorney.  Prior to attending law school he was an assistant professor at the University of Pittsburgh Medical School where he researched infectious diseases and vaccines.  He currently is in private practice and teaches Biotechnology law at the University of Pittsburgh law school.

Contact information:

C. Allen Black, Ph.D. Esq.:

TEL +1.412.908.3268;

email: [email protected];

www.patentlawyersite.com.

[Mises crosspost]

1.  For more on compulsory licensing, see Hickey, Kevin J.; Ward, Erin H., “The Role of Patents and Regulatory Exclusivities in Drug Pricing” (01/30/2024). [↩]