More on Fabry here: https://keionline.org/fabry
NIH rejects Fabrazyme March-In Petition
- December 7, 2010. Press release from Fabry patients: DHHS denies patient’s march-in request to end Genzyme’s rationing of treatment for Fabry Disease citing that FDA rules block manufactures from supplying the drug in a timely manner.
- December 7, 2010. Statements by KEI and others on NIH rejection of Fabrazyme March-In Request
- December 3, 2010, Letter from March L. Rohrbaugh, Director of the NIH Office of Technology Transfer to Allen Black. the attorney for Fabry patients.
- December 1, 2010 Determination by Francis Collins, Director of the NIH, rejecting the March-in Request.
- October 22, 2010. Letter from Mark Rohrbaugh to Amber Britton.
- August 27, 2010. AMSA, KEI, Public Citizen, PIRG and UAEM Request for an Expedited Hearing and Expanded Scope of Issues, Regarding Petition to Use Authority Under Bayh-Dole to End Rationing of Fabrazyme.
- July 15, 2014. KEI asks FTC to investigate Shire decision to abandon efforts to compete in US market for Fabry’s disease treatments
For additional context
- Public and Private Sector Research for Fabry’s Disease KEI Research Note 2010:2.
- Datapoints on Fabrazyme prices.
- The extension of US Patent No 5,356,804
- KEI web page on the Bayh-Dole Act, including links to other March-In cases.
- April 15, 2010, Andrew Pollack, Genzyme Drug Shortage Leaves Users Feeling Betrayed, New York Times.
- August 4, 2010, Richard Knox, “Fabry Patients Want Drug’s Patent Eased,” NPR
.
The Fabrazyme March-In
On August 2, 2010, Joseph M. Carik, Anita Hochendoner, and Anita Bova requested the Secretary of DHH to exercise Bayh-Dole march-in rights and grant an open license to use patents related to the manufacture of Fabrazyme® (agalsidase beta). The grounds for the request are that the patent owner and its exclusive licensee have harmed the public health by severely rationing the supply of agalsidase beta, the only approved therapeutic treatment for Fabry disease.
The lawyer for the petitioners is Alan Black. He has a web page on the complaint here.
As described in the petition to DHHS Secretary Kathleen Sebelius:
Joseph M. Carik, Anita Hochendoner, and Anita Bova are private individuals who have Fabry disease. They are prescribed Fabrazyme® to treat the disease, but they have not (and are not) receiving the prescribed dosage due the patentee’s and licensee’s inability to produce enough drug to treat all of the Fabry patients that have been prescribed Fabrazyme®. Their symptoms have worsened, and they are at greater risk of morbidity and death due to complications from the disease because of the severe and ongoing restriction in the supply of Fabrazyme®. Their position is identical to all Fabry patients because all patients are being rationed the drug by Genzyme.
Key Documents
- The August 2, 2010 cover letter to Secretary Sebelius.
- The August 2, 2010 Petition To Use Authority Under The Bayh-Dole Act To Promote Access To Fabryzyme® (Agalsidase Beta), An Invention Supported By And Licensed By The National Institutes Of Health Under Grant No. DK-34045.
Statement by KEI on Fabrazyme March-In Request:
“Persons who have Fabry’s disease are at risk today because of legal barriers to the competitive supply of agalsidase beta, marketed by Genzyme under the tradename Fabrazyme at a price of roughly $700 per day, or more than $250,000 per year. The Obama Administration officials who have the responsibility of approve or reject this petition will set a standard for the degree to which the holders of a patent on an NIH funded invention can be held accountable, when there are abuses of the patent monopoly. The petitioners seek the freedom to obtain independent competitive suppliers for this medicine. Genzyme has already earned billions of dollars on Fabrazyme. The NIH needs to end the legal monopoly, and permit greater competition in the supply of agalsidase beta. This will not only enhance the supply of this medicine, but it will send a message that the NIH will not tolerate abuses of patent rights for government funded inventions. Taxapyers and employers who pay for this expensive drug will also likely benefit in the longer term, as competition is expected to result in lower prices.” James Love, Director, KEI, 2 August 2010.
Reporting and commentary on the March-In Request
- August 2, 2010, Andrew Pollack, Patients Want Patent Broken on Genzyme Drug, New York Times
- August 3, 2010, Gareth Macdonald, “US patients call for Fabrazyme patent break” in-pharmatechnologist.com.
- August 3, 2010, Miles Wilson, “Patent to be broken on Genzyme drug?” Next Generation Pharmaceutical.
- August 3, 2010, Ed Silverman, “Patients Seek To Break Patent On Genzyme Drug,” Pharmalot.com
- Mike Masnick, August 3, 2010, “Patents Getting In The Way Of Saving Lives; Fabry Disease Sufferers Petition US Gov’t To Step In. from the patent-problems dept.” TechDirt.Com
- August 4, 2010, Richard Knox, “Fabry Patients Want Drug’s Patent Eased,” NPR.
- August 4, 2010, Bruce Lehr, “Should HHS Override Genzyme Fabrazyme Patents?” The Big Red Biotech Blog.
- August 4, 2010, George Miller, “Genzyme manufacturing woes yield patent threat,” FiercePharma Manufacturing
- August 4, 2010, Julie M. Donnelly, “Genzyme patients want patent lifted,” Boston Business Journal.
- August 4, 2010, “Fabry Patients Ask HHS to Issue Open License for Fabrazyme,” Drug Industry Daily, Vol. 9 No. 150. Subscription required.
- August 4, 2010, More Trouble at Genzyme, BioJobBlog.Com
- August 4, 2010, “Pharmakonzern schließt Werk – Patente verhindern Medikamentenproduktion“
- August 4, 2010, Patients Urge HHS to Widen Use of Fabry Drug Patent, BioWorld Today
- August 5, 2010, Victoria Slind-Flor, “Patients Ask Government to Open Genzyme’s Fabrazyme Patents,” Bloomberg.
- August 5, 2010, Nancy Faigen, “US activists petition NIH to break patent on Genzyme’s Fabrazyme,” Scrip
- August 5, 2010, Nirmal Choradia, “Fabry’s Patients Want Genzyme Patents Lifted,” BioSmash
- August 6, 2010, Patients Petition HHS to Break Exclusive Patent on Rare Disease Drug, AAU Weekly Wrap-up.
- August 9, 2010, Patients Petition HHS to Issue Open License for Fabrazyme, Washington Drug Letter, Vol. 42 No. 31
Reports on other aspects of the dispute
- April 15, 2010, Andrew Pollack, “Genzyme Drug Shortage Leaves Users Feeling Betrayed,” New York Times.
- April 21, 2010, Andrew Pollack, “Genzyme Expects a Fine of $175 Million,” New York Times.