November 2012 Archives

November 30, 2012

Orphan Drugs and Rare Diseases Spotlighted at New ACI Conference

Thumbnail image for Thumbnail image for Thumbnail image for Thumbnail image for Thumbnail image for Thumbnail image for aci_header_banner.gifOn November 28-29, 2012, the American Conference Institute ("ACI") held in Boston, Massachusetts, its inaugural conference: "Orphan Drugs and Rare Diseases: Maximizing Opportunities and Overcoming Stumbling Blocks in the Designation and Development Process". The Conference was well attended and featured a pre-conference boot camp on the Orphan Drug Act as well as a post-conference master class on overcoming clinical trial challenges and proving the safety and efficacy for orphan drugs.

One highlight of the conference was Timothy R. Cote, M.D., M.P.H., Principal, Cote Orphan Consulting (Director of FDA's Office of Orphan Drug Products Development (2007-2011)). Cote provided a back drop for how the Orphan Drug Act came about, emphasizing that while for each individual disease the afflicted patient population is small, when all of the orphan diseases are pooled together, actually the population is sizable and a rapidly-growing area. Dr. Cote described orphan drug development as a "high touch" field where the need for new therapies are often driven by families with afflicted members and typical "big pharma" strategies are less effective. Calling it "scrappy not crappy" science, Cote explained that unlike big pharma projects that involve thousands of patients and hundreds of millions of dollars to develop new therapies, orphan drugs often can be developed for under $10 million--in part because there are so few patients who are candidates for clinical studies and treatment. Cote said while approximately 60% of orphan drug designation requests are approved, the success stories are the products that obtain new drug application approval and win the highly-coveted seven-year exclusivity for the first orphan indication ("a horse race"). More importantly, the basic research required to find cures for orphan drugs often provides valuable medical knowledge in how our bodies work, which he called "pharma karma."

Another highlight of the conference was Christopher-Paul Milne, D.V.M., M.P.H., J.D., Associate Director, Tufts Center for the Study of Drug Development, Tufts University. Milne said that while the initial orphan market was thought of as relatively small, there are often higher rates of return than more "mainstream" diseases, particularly for diseases with more options for treatment. One phenomena that he tracked was the "ultra orphans," where the orphan population for treatment, which is restricted to less than 200,000 when applied for orphan drug designation in the United States, can get closer to the 200,000 number, and sometimes can expand to more mainstream numbers, if the therapy is later found to have non-orphan treatment options. While certain disease areas such as various cancers have good orphan drug representation, other therapeutic areas, such as neurology, have had less success stories, Milne reported. Milne added that amidst the tension of competition versus collaboration, it has often been important for unlikely partners to work together, with the help and motivation of patient groups, such as the National Organization for Rare Diseases ("NORD").

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November 29, 2012

FLH Partner Brian J. Malkin Speaks at ACI's Orphan Drugs and Rare Diseases on November 29, 2012 in Boston

Frommer Lawrence & Haug LLP Partner Brian J. Malkin will present on "Mastering the Intricacies of the Orphan Drug Designation Process: A Step-by-Step Guide to Navigating the Pathway" on November 29, 2012. Mr. Malkin's presentation is part of ACI's inaugural conference, "Orphan Drugs and Rare Diseases: Maximizing Opportunities and Overcoming Stumbling Blocks in the Designation and Development Process" in Boston, Massachusetts from November 28-29, 2012. Mr. Malkin's presentation will include how to assess whether a compound/indication qualifies for orphan drug designation, what benefits may be achieved by obtaining an orphan drug designation prior to product/indication approval, and how to respond to a rejection for an orphan drug designation request, including demonstrating clinical superiority. For more information, see here.

November 26, 2012

Cumberland Pharmaceuticals Sues FDA After Denial of Citizen Petition and Approval of ANDA

FDA logo.jpgOn November 13, 2012, Cumberland Pharmaceuticals Inc. ("Cumberland") sued FDA, the Commissioner of Food and Drugs, and the Secretary of Health and Human Services in federal district court for the District of Columbia for denying Cumberland's Citizen Petition and approving InnoPharma, Inc.'s ("InnoPharma's") abbreviated new drug application ("ANDA") for an acetylcysteine injection. Cumberland asserted that the denial of the Citizen Petition and the approval of InnoPharma's ANDA were "arbitrary, capricious, and abuse of discretion, and not in accordance with law, all in violation of the APA [Administrative Procedure Act]" and "violated the Federal Food, Drug, and Cosmetic Act ('FDCA')," according to its complaint.

Cumberland holds the approved new drug application ("NDA") for the reference listed drug ("RLD"), Acetadote®, indicated to prevent or lessen hepatic injury in adults and children when administered intravenously within 8 to 10 hours after ingestion of a potentially hepatotoxic quantity of acetaminophen. The original formulation of Acetadote® contained edentate disodium ("EDTA") as a chelating agent that was believed to be necessary for the stability of the product. FDA approved Acetadote® in January of 2004, but conditioned its approval on Cumberland's agreement to perform post-approval studies to evaluate whether EDTA could be removed from the formulation in view of safety concerns. EDTA has been associated with a significant drop in serum calcium levels, which may result in fatality, hypokalemia, hypomagnesaemia, or hypotension as well as allergic reactions.

As a result, Cumberland developed and obtained approval in January 2011 from FDA for a new EDTA-free formulation after, according to the complaint, surprisingly demonstrating that EDTA was unnecessary to maintain drug stability. Cumberland then withdrew the EDTA-containing formulation from the market.

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November 21, 2012

ACI's Legal, Regulatory and Compliance Forum on Orphan Drugs Boasts a "Who's Who" of FDA Regulatory and Legal Experts

Thumbnail image for Thumbnail image for Thumbnail image for Thumbnail image for Thumbnail image for aci_header_banner.gifAmerican Conference Institute (ACI) will be holding its inaugural Legal, Regulatory and Compliance Guide to Orphan Drugs and Rare Diseases in Boston at the Hyatt Regency on November 28-29, 2012. This unique program features a distinguished faculty of over two dozen leading legal and regulatory orphan drugs and rare diseases experts - including the former director of FDA's Office of Orphan Products Development, senior in-house pharmaceutical counsel, leading patient advocates, and preeminent FDA and patent attorneys - who will address the intricacies of the FDA's orphan drug designation process as well as the challenges affecting orphan drug development and commercialization and overall patent portfolio considerations.

The ACI conference provides a comprehensive forum for the industry to discuss the latest legal and regulatory developments affecting orphan products and therapies for rare diseases and to analyze the strategic considerations in drug development aimed at providing patients facing an unmet medical need with safe and effective life changing therapy. In light of recent cases concerning FDA enforcement of orphan exclusivity, the conference will also feature a spotlight session on "Protecting Orphan Drug Designation and Proactively Guarding Against Potential Liability."

More information about this event, including a full agenda, faculty list, and brochure can be accessed at

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November 19, 2012

Jazz's Xyrem® Citizen Petition Denied Yet Another Remains Pending

sleepingperson.pngLast week, FDA denied a Citizen Petition filed by Jazz Pharmaceuticals, Inc ("Jazz"). The May 18, 2012 Petition concerned bioequivalence studies relating to Xyrem® (sodium oxybate), Jazz's oral solution indicated for the treatment of excessive daytime sleepiness and cataplexy in patients with narcolepsy. Before issuing its decision, FDA received and considered public comment from Roxane Laboratories ("Roxane"), who had an Abbreviated New Drug Application ("ANDA") referencing Xyrem accepted for review in late 2010.

Jazz asked FDA to take three actions. First, Jazz asked FDA to immediately publish in The Orange Book bioequivalence requirements specifying whether in vitro or in vivo bioequivalence studies, or both such studies, are required for ANDAs referencing Xyrem®. Jazz claimed that FDA's failure to have done so within the first 30 days of Xyrem®'s approval was a violation of the Federal Food, Drug, and Cosmetic Act ("FD&C Act") (See 21 U.S.C. § 355(j)(7)(A)(i)-(ii).) and the Administrative Procedure Act ("APA") (See 5 U.S.C. § 706.). FDA disagreed; the Agency found several flaws with Jazz's arguments. First, requiring the publication of bioequivalence data type for ANDAs within 30 days of new drug approval would be inconsistent with other sections of the FD&C Act, as well as certain FDA regulations. Second, adopting Jazz's arguments would require the Agency to generate and evaluate the scientific data needed to understand bioequivalence characteristics at the time the reference listed drug ("RLD") was approved. FDA disfavored this position, because it would prevent FDA from gaining insight into the characteristics of the RLD during its marketing. Many products never face generic competition, or only do so after the development of acceptable bioequivalence methodologies. As such, FDA reasoned it would be a waste of Agency resources to determine what types of bioequivalence studies are needed within the first 30 days of the RLD's approval. Third, there is no indication Congress meant the statute to require what Jazz sought, and no court has construed the statute as requiring as much. Finally, FDA noted that Jazz's interpretation would actually prejudice those who the statute was meant to protect, i.e., the ANDA sponsors. Requiring FDA to publish bioequivalence requirements within 30 days of the RLD approval would diminish FDA's ability to provide ANDA sponsors with information about the best ways to demonstrate bioequivalence.

Next, Jazz asked FDA not accept for review, review, or approve any ANDA referencing Xyrem® unless and until FDA has published bioequivalence requirements in the Orange Book specifying whether in vitro bioequivalence studies, in vivo bioequivalence studies, or both such studies, are required for ANDAs referencing Xyrem®. Jazz argued that to do so would violate the APA. The drug company reasoned that an ANDA must reference an RLD, and an RLD does not become an RLD until FDA issues the bioequivalence requirements. As such, Jazz concluded that an ANDA can only be accepted for review after FDA issues bioequivalence requirements. Accordingly, Jazz asked FDA to set aside its acceptance of Roxane's ANDA because this was "agency action . . . without observance of procedure required by law," "not in accordance with law," "in excess of statutory jurisdiction, authority, of limitations," and "short of statutory right." Again, FDA declined. The Agency claimed that refusing to accept an ANDA on those grounds would conflict with various FD&C Act sections, including Section 505(j)(2)(A), which delineates what information is required in an ANDA and does not list the bioequivalence requirements, as well as certain FDA regulations. It also refused to accept an interpretation of the statute that would punish ANDA applicants, even if in compliance with the statutory requirements, for FDA's failure to publish the bioequivalence requirements.

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November 16, 2012

American Conference Institute's 2nd Comprehensive Guide to Patent Reform

Thumbnail image for Thumbnail image for Thumbnail image for Thumbnail image for aci_header_banner.gifAmerican Conference Institute's 2nd Comprehensive Guide to Patent Reform once again unites experienced in-house counsel from top innovators, private practice experts, and senior officials from the USPTO to answer patent professionals' most pressing questions, including:

  • What does "first to file" actually mean under the AIA requirements? Which system can you or should you file under - the current first to invent or the new first to file (or both)? And how do you avoid first-to-file bubble filings before 3/15/2013?
  • When can on-sale and public use activity be considered prior art? Has secret §102(f) prior art been eliminated?
  • Do you need to include best mode in the application or not and what happens if you don't? Is best mode completely toothless now? How will examiners be able to address the best mode issue?
  • What will be required in the PGR process? What type of discovery? Expert witnesses? How do the estoppel provisions alter your analysis of whether to engage in the PGR system?
  • What estoppel provisions are associated with IPRs and how are these different from the inter partes reexamination provisions? When do you file a 3rd party parallel IPR?

  • For more information, please visit our website:

    FDA Lawyers Blog subscribers are entitled to a discount when referencing the code: FDA 200.

November 12, 2012

FLH Attends ITC Trial Lawyers Association Annual Meeting in Washington, D.C.

Thumbnail image for ITC.bmpOn November 7, 2012, FLH Partner Brian J. Malkin and Associate Christopher Gosselin were in attendance at this year's International Trade Commission Trial Lawyers Association Annual Meeting at the International Trade Commission ("ITC") in Washington, D.C. The theme of this year's meeting was the enforcement of ITC exclusion orders at the border, and the relationship between customs and the ITC.

ITC Chairman Irving A. Williamson opened the meeting, stressing his commitment to reducing the overall length of Section 337 Investigations from 16 months to 13.5 months. He also promised to continue efforts to reign in the cost of discovery during Section 337 Investigations.

During the refreshment break, guests were invited to view the new courtroom on the second floor, and heard brief remarks from Chief Administrative Law Judge ("ALJ") Charles E. Bullock. The new courtroom is spacious and well equipped with modern technology ranging from video projection screens to LCD monitors at every table. The courtroom should see plenty of use once it opens for business, as even with the new courtroom, the ITC still has more ALJs than available space. Although the ITC saw a 30% decrease in newly instituted investigations in 2012 after a record setting number in 2011, the ALJs continue to manage a near record level of active investigations.

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November 9, 2012

K-V Not Done Fighting for Makena®'s Orphan Drug Rights Over Compounding Pharmacies

gavelgold.jpgK-V Pharmaceutical Company ("K-V") has taken the fight over its preterm-birth prevention drug, Makena® (17-hydroxypreogesterone caproate solution) ("HPC"), to the International Trade Commission ("ITC"). In its complaint, K-V asks the ITC to: (1) issue a temporary general exclusion order prohibiting any unauthorized importation of HPC and (2) issue a temporary cease and desist order stopping owners, importers, and consignees from importing, selling, offering for sale, distributing, or soliciting any HPC unless authorized by K-V.

K-V states that its merits argument will focus on the importation of HPC for the purpose of making compounded versions of Makena®. K-V argues that importation of HPC for use in compounding copies of Makena® is a violation of Section 337 of the Tariff Act of 1930. According to Section 337, "[u]nfair methods of competition and unfair acts in the importation of articles . . . in the United States, or in the sale of such articles by the owner, importer, or consignee, the threat or effect of which is . . . to destroy or substantially injure an industry in the United States," are "unlawful." 19 U.S.C. § 1337(a)(1)(A). K-V argues that, because all HPC in the United States comes from abroad, and the only use for HPC is in the manufacture of Makena® or allegedly unlawful copies of Makena®, the importation of HPC undermines federal law and K-V's statutory orphan drug exclusivity and "is clearly an unfair act and an unfair method of competition."

According to the Complaint, all the requisites for temporary relief are present. First, there will be immediate and irreparable harm to K-V because the pharmacies compounding copies of Makena® have diverted so much potential revenue from K-V that the drug company has had to file for Chapter 11 bankruptcy. Second, as mentioned above, there is a likelihood of success on the merits. K-V argues that the importation of HPC for the use in compounding violates the prohibition against mass-scale compounding and undermines K-V's statutory orphan drug exclusivity. These unfair acts and unfair methods of competition are threatening to destroy K-V, i.e., the domestic industry. Third, K-V argues that the following public interest factors favors granting the desired relief: (1) prevention of preterm births; (2) ensuring safe and effective drugs; (3) preservation of a domestic industry; and (4) effectuating Congress's intent to promote development of treatments for rare conditions. Finally, K-V states that it faces the balance of hardships. K-V argues that the compounding pharmacies have few, if any, protectable rights, whereas K-V has a "Congressionally-granted seven-year exclusive right to market Makena." Additionally, K-V notes that its business's survival depends on the success of Makena®, whereas the proposed respondents compound many other products and will survive if they are unable to compound Makena® during the exclusivity period.

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November 5, 2012

En Banc Review of the Standard for Reviewing USPTO Claim Constructions Recommended by Panel of Federal Circuit Judges

Thumbnail image for Thumbnail image for Thumbnail image for federalcircuit.jpgA panel of three federal circuit judges, Judges Newman, Plager, and Wallach, upheld a decision of the Board of Patent Appeals and Interferences ("Board") in an inter partes reexamination rejecting certain claims of U.S. Patent No. 6,721,178, owned by Flo Healthcare Solutions, LLC ("Flo"), in an opinion decided on October 23, 2012. U.S. Patent No. 6,721,178 claims a mobile computer workstation for the medical profession.

While the judges disagreed with the reasoning of the Board, the conclusion by the Board was upheld. Judge Plager wrote in the opinion that "the Board erred in holding that the 'height adjustment mechanism' limitation of the claims invokes 35 U.S.C. § 112, ¶ 6" because the term "means" was not recited in the claims and the limitation contains a term that designates structure. However, the Board correctly held that "the claims do not require a length-adjustable vertical beam" since the claims do not recite such a limitation even though Flo had the opportunity to amend the claims to include this limitation. Thus, the panel agreed that "the prior art rejections must stand in the absence of such a limitation."

Notably, Judge Plager and Judge Newman both wrote additional views to this opinion urging the en banc review of the standard used by the Federal Circuit when reviewing claim construction decisions by the Board. Both judges were concerned that the Federal Circuit had announced conflicting standards for reviewing such claim constructions. This review would provide a clear standard for the PTO and inventors urged the judges.

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November 2, 2012

Gore Asks Supreme Court to Review Joint Inventorship Standard

Thumbnail image for Thumbnail image for Supreme Court.jpgOn October 12, 2012, W.L. Gore & Associates Inc. ("Gore") filed a petition for writ of certiorari with the U.S. Supreme Court, asking the High Court to review the Federal Circuit's denial of joint-inventor status to one of Gore's engineers. The patent-in-suit, U.S. Patent No. 6,436,135 ("the '135 patent") lists Dr. David Goldfarb ("Goldfarb") as the sole inventor (Bard is the assignee.), and Gore argues that Peter Cooper ("Cooper"), head of Gore's vascular prosthetic research program, should be listed as a joint inventor. Gore alleges that the Federal Circuit erred in setting forth a new standard for joint inventorship that directly conflicts with legislative intent and the plain text of the Patent Act.

In the district court, the jury held that Gore willfully infringed Bard's patent, and the judge doubled the jury's damage award, granting $371 million to Bard. The Federal Circuit affirmed the district court's decision in February 2012, and Gore filed a combined petition for panel rehearing and rehearing en banc. In June 2012, the Federal Circuit granted the motion for rehearing en banc, "for the limited purpose of authorizing the panel to revise the portion of its opinion addressing willfulness." Gore then filed the instant petition.

In the petition, Gore alleges that in 1971, it began a research program focused on the use of stretched Teflon® ("Gore-Tex®") in vascular prosthetics, or artificial blood vessels. Gore created implantable vessels with varying features, and hired doctors to implant the vessels and to report which ones were successful. Cooper then started an additional experiment involving the porousity of the tubes. Cooper sent small, medium, and large-pore tubes to the physicians, and the results showed that the large-pore tubes (which had a greater "internodal" distance" or "fibril length") were the winners. Based on this, Cooper conceived of the instant invention on May 1, 1973.

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