[Ip-health] BNA: NIH Declines to Exercise 'March-In Rights' For Fabrazyme Patent; Patients to Appeal

Judit Rius Sanjuan judit.rius at keionline.org
Fri Dec 17 11:50:00 PST 2010


Medical Research Law & Policy Report®
Volume: 9 Number: 24
December 15, 2010

NIH Declines to Exercise 'March-In Rights' For Fabrazyme Patent; Patients to Appeal

Although the National Institutes of Health expressed great concern about the effect of the rationing of the drug Fabrazyme on the health of patients with Fabry disease, the agency Dec. 3 denied the patients' request that it exercise its “march-in rights” under the Bayh-Dole Act and break Genzyme Corp.'s patent hold on the drug.

In a letter to Attorney C. Allen Black Jr., who filed the patients' petition, Mark L. Rohrbaugh, director of the NIH Office of Technology Transfer, wrote that the agency remained “very concerned about the health of Fabry patients and their access to Fabrazyme” and had not “closed this request.” However, NIH said it had determined not to exercise its march-in authority because “any licensing plan that might result from such a proceeding would not, in our judgment, remedy the current problems associated with the access to Fabrazyme.”

The Bayh-Dole Act allows any “responsible applicant” to request march-in rights under 35 U.S.C. §203, which empowers the federal agency funding the research to “march in” and issue patent licenses on its own when the holder of a patent that resulted from the research is not “reasonably” satisfying U.S. health or safety needs. The petitioners are representatives of the entire class of patients with Fabry disease, a rare, X-linked recessive (inherited) disease that interferes with fat metabolism and causes kidney and heart problems that can lead to death. The patients noted in their Aug. 3 petition that Fabrazyme was the direct result of an NIH grant to the Mount Sinai School of Medicine.

Genzyme produces Fabrazyme under an exclusive license from Mount Sinai. According to the petition, although Genzyme's initial production of Fabrazyme was sufficient to meet the needs of all U.S. Fabry patients, Genzyme decreased production in mid-2009 as a result of a viral infection in its Allston, Mass., plant. FDA initiated action against Genzyme that resulted in a May consent decree that required the drugmaker to correct the Allston plant quality violations and to disgorge $175 million in unlawful profits from the sale of products made at Allston.

Genzyme Nov. 24 announced that it had ended fill/finish operations within its Allston plant, as required by the FDA consent decree. Fill/finish activities for all drugs produced at the plant, including Fabrazyme, for the U.S. market now take place at Genzyme's Waterford, Ireland, plant and at an external contract manufacturer, according to the announcement.

No March-In, but Door Not Closed.

Attached to the letter to Black was a determination memorandum signed by NIH Director Francis S. Collins, dated Dec. 1, which concluded that the regulatory hurdles that would be necessary for another manufacturer to overcome to be able to produce Fabrazyme would make NIH's exercise of its march-in rights futile.

The memorandum noted that a manufacturer must procure clinical materials, gather preclinical data, and ensure the safety of a product before the Food and Drug Administration will approve an investigational new drug (IND) application to authorize the beginning of clinical trials. Following clinical trials, FDA must approve a biologic license application (BLA) for a biologic drug, and that review can take 10 months at a minimum. Even with a license to the Genzyme patents, NIH determined FDA approval simply would take too much time. NIH also has indicated it had not received any information that suggested a qualified third party was ready to supply an effective, alternate therapy.

The memorandum noted that Genzyme expects the production of Fabrazyme to be back to full levels in the first half of 2011, which should address the shortage. NIH agreed to monitor the situation and requested that Mount Sinai provide monthly reports on Genzyme's manufacturing process. The memorandum indicated that NIH would reconsider its opinion upon receiving any unfavorable updates.

Bayh-Dole Without Remedy?

Three previous requests to NIH to exercise these rights for other drugs have been denied, but the Fabrazyme petitioners attempted to distinguish those cases from theirs. NIH did not reference those decisions in its memorandum of determination.

Black told BNA Dec. 10 that the petitioners intend to appeal NIH's decision on both procedural and substantive grounds. First, he said, there should have been a notice-and-comment period for the petition, but there was not. Second, Black said, “What the decision indicates is that the Bayh-Dole Act really has no remedy anymore.”

He continued, “The U.S. Department of Health and Human Services is supposed to reconcile the [Federal] Food, Drug, and Cosmetic Act with the Bayh-Dole Act, and it controls both NIH and FDA. And yet in its memorandum NIH effectively says, sorry, our sister agency is too slow and there's nothing we can do about it. We think that creates a substantive problem with the Bayh-Dole Act.”

By John T. Aquino

The NIH letter can be found at http://op.bna.com/hl.nsf/r?Open=jaqo-8bzlzr.

The NIH memorandum of determination is at http://op.bna.com/hl.nsf/r?Open=jaqo-8bzm2z.

Copyright 2010, The Bureau of National Affairs, Inc.

Judit Rius Sanjuan
Knowledge Ecology International (KEI)
NYC Phone: 212 222 5180
Washington DC Phone: 202 332 2670

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