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February 19, 2014

Leerink's Global Healthcare Conference 2014 Focuses on a Variety of Emerging Health Products

leerink.pngOn February 12 and 13, 2014, Leerink Partners LLC ("Leerink") held its annual Global Healthcare Conference at the Waldorf-Astoria Hotel in New York, New York. Each year the Conference includes emerging themes in healthcare, where Leerink's equity analysts moderate discussions with MEDACorp specialists to provide unique and timely insights.

In addition to the company presentations, this year's line up featured the following panels or keynote speakers with some observed comments or trends:

• Panel: The Future of Medical Devices in an Evolving Landscape: A Shifting Emphasis to Patient Monitoring and Customizable Solutions

  • Patients view surgeons that incorporate robotics in their practice as the better doctors, driving more surgeons to utilize them in their practice. As surgeons become more familiar with these devices, patients may have more options for surgical procedures and implants.

  • Larger companies are looking at controlling infections caused by implanted medical devices with special coatings--either anti-infectives or antibiotics, particularly for use in higher-risk patients. There is an increasing need, however, for implants to have built-in tools for monitoring the devices. But as medical devices become more complex, such as hip, knee, or total joint replacements, these devices will require preapproval marketing applications ("PMAs") with clinical data rather than less costly and time-consuming 510(k)-type premarket clearance applications. Since PMAs cost companies more than 510(k) applications, these newer devices will cost third party payors and patients more.

  • Hospitals continue to be under a lot of pressure not to lose patients, so they may seek lower margins by having surgeons add anti-infective coatings or antibiotics rather than purchasing more costly versions with the coatings or by importing "generic" implants from other countries that may not be as rugged as the versions cleared for use in the U.S. FDA's new unique device identifiers and improved monitoring, however, may reduce use of such imported devices with unclear pedigrees.

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January 14, 2014

Biosimilar Naming the Subject of Recent J&J Citizen Petition

Jnj.jpgJohnson and Johnson ("J&J") recently joined the discordant chorus of stakeholders and commentators who have weighed in on the issue of naming for biosimilar products. On the one hand, some advocate for shared International Non-Proprietary Naming ("INN") system names between a biologic approved under Section 351(k) of the Biologics Price Competition and Innovation Act of 2010 ("BPCIA") and the reference protein product ("RPP"). Conversely, others argue that biosimilars and RPPs should be assigned unique INNs. Whether biosimilar products are given the same or unique names matters: biosimilar products with unique names will likely require independent marketing and detailing (i.e., automatic substitution will not be available). For its part, J&J requests that FDA "require biosimilars to bear nonproprietary names that are similar to, but not the same as, those of their reference products or other biosimilars."

J&J cites to its experience with Eprex®/Erypo® recombinant human erythropoietin (epoetin alfa), to inform its position on biosimilar naming. In particular, J&J identified four considerations that arose from its experience: (1) reliable pharmacovigilance mechanisms are necessary for postmarket safety; (2) products may undergo clinically-meaningful changes over time; (3) effective pharmacovigilance can only occur when it is possible to identify the product administered to a patient; and (4) switching products can interfere with determining which product is responsible for any given adverse effect. For example, J&J received reports of erythopoetin antibody-mediated pure red cell aplasia in Thailand between 2004-2007 but were unable to pinpoint the adverse event reports to a specific epoetin product due to incomplete documentation and frequent product switching.

Based on this experience, J&J argues that giving a biosimilar product the same name as the RPP would interfere with pharamacovigilance. For example, J&J states that, "to the extent that adverse event reports identify a product solely by nonproprietary name, shared names would complicate if not prevent tracing a safety signal to a specific product." J&J also states that physicians may submit adverse event reports that incorrectly identify the responsible product if switching occurs without the knowledge of the physician.

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January 2, 2014

Cellular and Gene Therapy Advisory Committee Meeting Rescheduled for February 2014

genetherapy.jpgOn February 25-26, 2014, FDA will hold a meeting of the Cellular, Tissue, and Gene Therapies Advisory Committee. A majority of the meeting will concern oocyte modification in assisted reproduction for the prevention of transmission of mitochondrial disease or treatment of infertility. While FDA will post meeting materials at least two business days before the meeting, one item that will likely be discussed is a workshop held in September 2002 on Evidence Based Assisted Reproductive Technologies (ART) that concerned oocyte modification. Approximately half of the second day will focus on considerations for the design of early-phase clinical trials of cellular and gene therapy products, which was the topic of a revised guidance published on July 2, 2013. This meeting was originally scheduled for October 22-23, 2013 but was postponed "due to resource constraints arising from the government shutdown."

The July Draft Guidance provided recommendations to assist in designing early-phase clinical trials of cellular therapy ("CT") and gene therapy ("GT") products, collectively referred to as "CGT products", which covers most Phase 1 trials and some Phase 2 trials. FDA considers clinical study designs for CGT products to be different because of the way the products work and the potential for substantial risk. In the past, FDA halted CGT therapies due to experiences that included: (1) multiple-organ failure and death of a subject receiving a GT product for ornithine transcarbamylase deficiency, (2) late-onset T-cell leukemia in subjects who received a GT product for X-linked severe combined immunodeficiency, and (3) the development of tumors in the brain and spinal cord of a patient who received intrathecal allogenic stem cells for ataxia telangiectasia.

FDA's Guidance on early-phase clinical trials explained that unlike many small molecule drugs, there is much less experience across a broad population with CGT products, leading to more uncertainty with clinical study design and controls. Some CGT products persists in humans for an extended time period and the administration may involve surgery or other invasive procedures that may require use of an investigational medical device. Allogenic CT products, GT vectors, and proteins that may be produced by CGT products have the potential to produce an immune response that may produce an unintended adverse reaction or sensitivity to a CGT product in the future. CGT products are cellular products and so mimic the complex, dynamic nature of living cells, which can migrate within the recipient's body.

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November 11, 2013

Partially Hydrogenated Oils Tentatively No Longer GRAS

transfat.jpgFDA has tentatively found that partially hydrogenated oils ("PHOs"), the primary dietary source of trans fatty acids, are no longer generally regarded as safe ("GRAS"). While PHOs are used in a number of food products (e.g. margarine, shortening, and baked goods), they have been linked to significant health risks, such as coronary heart disease. FDA's tentative determination that PHOs are no longer GRAS means that PHOs would now be classified as "food additives," subject to Section 409 of the Federal Food, Drug, and Cosmetic Act ("FD&C Act") (21 U.S.C. 348). FDA does not allow food manufacturers to sell food additives, directly or indirectly, without prior approval for us by FDA.

The FD&C Act defines a "food additive" as "any substance the intended use of which results or may reasonably be expected to result, directly or indirectly, in its becoming a component or otherwise affecting the characteristics of any food...." 21 U.S.C. 321(s). Except that GRAS substances are not considered food additives. A substance is GRAS if it is generally recognized to be safe under the conditions of its proposed use "among experts qualified by scientific training and experience to evaluate its safety, as having been adequately shown through scientific procedures." In addition, if the substance had been used in food before January 1, 1958, it could be considered GRAS based on experience form its "common use" in food. However, even common use in food cannot overcome new evidence demonstrating that a consensus no longer exists that the substance is safe.

While some GRAS substances are listed in the regulations, there is no comprehensive list of GRAS substances. With the passage of the Food Additives Amendment to the FD&C Act in 1958, FDA established a list of GRAS substances. And in 1972, FDA instituted a notice-and-rulemaking procedure for affirming certain substances as GRAS. However, in 1997, FDA instituted a voluntary notification program for GRAS substances, which does not require notice-and-comment rulemaking. Thus, in many cases, food manufacturers and users have been responsible for determining whether substances are GRAS in light of the views of experts. For example, partially hydrogenated soybean oil and partially hydrogenated cottonseed oil were considered GRAS based on common use prior to 1958, but are not listed as GRAS in any FDA regulation.

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October 4, 2013

Biotech Giants Provide Insight to Going Public at Tech Council of Maryland Breakfast in Bethesda

biotechgiant.jpgOn October 3, 2013, the Tech Council of Maryland ("Tech Council") hosted a Bioscience Series presentation "Industry Giants" in Bethesda, Maryland. The Tech Council's is Maryland's largest trade association for bioscience and technology companies employing more than 200,000 in the region. The Industry Giants event was kicked off by the Tech Council's new Chief Executive Officer, Philip Schiff, formerly chief strategy officer of the American Association of Blood Banks ("AABB") (also known as "Advancing Transfusion and Cellular Therapies Worldwide", which is a not-for-profit trade association based in Bethesda, Maryland, that that advances the practice and standards of transfusion medicine and cellular therapies to optimize patient and donor care and safety.)

The Industry Giants panel was moderated by Ray Briscuso, Annual Conference Chief Executive AdvaMed, former Executive Director, Biotechnology Industry Organization ("BIO"), and featured speakers: Steven Dubin, former Chief Executive Officer of Martek Biosciences ("Martek"); Charles Fleischman, former President, Chief Operating Officer and Chief Financial Officer of Digene Corporation ("Digene") (now Qiagen), and Tom Watkins, former Chief Executive of Human Genome Sciences ("HGS"). All three former executive speakers came from companies that were acquired for over a billion dollars yet have managed to maintain their ties to the life sciences field.

Dubin's Martek focused on a microbial platform with a unique technology for nutrition and functional ingredients. Dubin recounted how it took a long time for the company to become profitable (18+years), which he thought would be a hard model to follow in today's times, particularly since they went public at least 6-7 years before there was a profit. Dubin thought that he learned by getting involved in the biotech community and by bringing in people who he thought were smarter than he was to help build passion in the company culture, which he found critical. He thought that emerging biotechnology companies should not focus so much on the exit strategy and found going public a "huge distraction" for the company to continuously answer to its investors. When asked what he respected from individuals looking to provide services for his company, he said that he thought it was important to first build a relationship and understand the company's problems before asking for work.

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September 27, 2013

Strategic Scientific Business Tactics Explored at MassBio Forum in Cambridge

biotechnology.jpgOn September 26, the Massachusetts Biotechnology Council ("MassBio") hosted the fourth part of its "Adventures in Biotech" Forum series entitled, "Executing the Dream II: Eyes on the Price - Strategic Science Tactics & the Pursuit of Business Objectives" . The purpose of this Forum was to discuss ways that scientific strategy may be worked into the philosophy of a new company, how the scientific strategy can propel the growth of the company, and how the scientific strategy may change over time as a result of that growth. The speakers included Birgit Schoeberl, Vice President, Early Stage Discovery, Merrimack Pharmaceuticals ("Merrimack"); Muthiah Manoharan, Ph.D., Senior Vice President Drug Discovery, Alnylam Pharmaceuticals ("Alnylam"); Laurence Reid, Ph.D., Senior Vice President, Chief Business Officer, Alnylam; and Steven Tregay, Ph.D., Founder, President and Chief Executive Officer, Forma Therapeutics ("Forma").

Each of the speakers had a different approach as to how they incorporated their scientific strategy into their business. Tregay, for example, described Forma as a "sustainable research and development discovery engine" focused on oncology therapies that can screen 30-40 individual (not combinational) targets a year using an iterative process that includes: computational and medicinal chemistry, parallel synthesis, X-ray crystallography and relevant biology studies. According to Tregay, Forma's science permits it to mitigate risk by running in parallel a large number of discovery programs while continuously assessing the molecules, prioritizing targets for their pipeline at the appropriate scale, and maintaining communication between all scientists, and project teams. As part of its strategy, Forma first goes broad to investigate a cancer target technology and then more specific as aspects of that technology are validated and ready for candidate screening. Integral in Forma's strategy is forming partnerships with companies interested in screening many potential cancer candidates quickly rather than few candidates exhaustively, where it already has formed partnerships with Celgene, Boehringer Ingelheim, Genentech, and Johnson & Johnson, as well as some research universities.

Reid explained that Alnylam's focus has been on building its platform technology on ribonucleic acid interference ("RNAi"), a natural mechanism for silencing specific genes. RNAi is a biological process in which ribonucleic acid ("RNA") molecules inhibit gene expression, typically by causing the destruction of specific messenger RNA ("mRNA") molecules. RNAi has gone by other names in the past, including co-suppression, post transcriptional gene silencing ("PTGS"), and quelling. Genes provide cells with the instructions for making proteins, and abnormal proteins are frequently the cause of human disease. The theory behind RNAi therapy, therefore, is to use double-stranded RND ("dsRNA") to silence certain genes involved in the diseased state and reduce the occurrence of the associated disease. Scientists at Alnylam developed a new strategy to trigger RNAi in mammalian cells using relatively-small dsRNAs--long enough to induce RNAi, but small enough to avoid inducing an immune response--to permit RNAi to be considered as a new therapeutic strategy.

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August 21, 2013

GSK Flulaval® Quadrivalent Vaccine Receives FDA Approval

boywithflu.jpgOn June 28, 2013, FDA approved GlaxoSmithKline Biologicals' ("GSK") Flulaval® Quadrivalent (Influenza Virus Vaccine) for the 2013-2014 flu season. Flulaval® Quadrivalent protects against two influenza-A virus strains and two influenza-B virus strains and is approved for ages 3 and older. This season is the first time that vaccines protecting against more than three influenza strains will be commercially available. The other quadrivalent vaccines that are currently available include: Astrazeneca/Medimmune's FluMist® Quadrivalent, GSK's Fluarix Quadrivalent, and Sanofi's Fluzone® Quadrivalent.

Seasonal flu is caused by either type-A influenza strains or type-B influenza strains. Each year, the World Health Organization recommends that vaccines address the two type-A strains that are most common in humans, and the type-B strain that is expected to be the most predominant in the upcoming flu season. Vaccines that protect against three influenza strains are referred to as trivalent vaccines. "Trivalent influenza vaccines have helped protect millions of people against flu, but in six of the last 11 flu seasons, the predominant circulating influenza B strain was not the strain that public health authorities selected," said Dr. Leonard Friedland, V.P. and Head, GSK North America Vaccines Clinical Development and Medical Affairs. Dr. Friedland also noted that, "Trivalent vaccines do reduce influenza risk even in years when a vaccine strain-mismatch occurs, though quadrivalent influenza vaccines are the important next step in broadening strain coverage." For the 2013-2014 flu season, the World Health Organization recommended protection against a second type-B influenza strain.

Three other quadrivalent vaccines have been approved by FDA for the 2013-14 flu season. Fluarix Quadrivalent is an intramuscular vaccine that requires 1-2 doses depending on a person's age and/or vaccination history. It is approved for ages 3 and older. FluMist® Quadrivalent is a live, attenuated vaccine that is administered via intra-nasal spray in 1-2 doses depending on a person's age and/or vaccination history. It is approved for ages between 2 and 49 years. Fluzone® Quadrivalent is an intramuscular vaccine that requires 1-2 doses depending on a person's age and/or vaccination history. It is approved for ages 6 months and older.

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April 29, 2013

The Venture Café Hosts Life Sciences Week - FLH Partner Brian J. Malkin Holds Office Hours 3-5pm

Thumbnail image for venturecafe.jpgThis week The Venture Café in Cambridge, Massachusetts hosts Life Sciences Week with a special focus on life science research in the Greater Boston area. Featured life science research speakers include Akrivis (ADAPT™ technology, which allows cancer detection much earlier by increasing the sensitivity of early biomarker detection and enabling the medical imaging of millimeter-sized cancer tumors), Human Metabolome Technologies (metabolome profiling for tumor metabolism, biomarker discovery and production optimization), Advirna (powerful and innovative tools to regulate activity of genes inside living cells), Heartbt Foundation (newly-developing non-profit seeking to boost translational research by bridging medical, professionals, big pharma, researchers, NGOs, the general public, and others), Extend Biosciences (startup biotech company with a platform technology that enables the development of long-acting peptide based drugs), Anchor Therapeutics (pre-clinical stage drug discovery company advancing Pepducin Technology, a novel approach towards allosteric modulators of G-protein coupled receptors), Lab Central (soon-to-open facility to house up to 65 scientists, in 20 individual lab stations and 9 private lab suites in the heart of Kendall Square), and Cellanyx (biomarker-based diagnostic biopsy test to determine oncogenic and metastatic potential for prostate tumors). The Venture Café will also host a Roundtable of Life Sciences Entrepreneurship in Massachusetts, featuring Peter Abair of the Massachusetts Biotechnology Council ("MassBio"), Pamela Norton from the Mass Life Sciences Center, and Peter Parker of Lab Central.

During Life Sciences Week, FLH Partner Brian J. Malkin will host Office Hours from 3-5pm EST along with several other service providers in the healthcare field. Mr. Malkin's Office Hours description reads:

Chat with Brian J. Malkin, Partner at Frommer Lawrence & Haug LLP. Brian specializes in FDA-regulated products, in particular pharmaceutical, biotechnology products and biosimilars. Discuss intellectual property, pathways for FDA approval, as well as life cycle management and due diligence investigations. Brian frequently speaks on a variety of IP- and FDA-oriented topics, and is editor of the FDA Lawyers Blog.

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April 23, 2013

FLH Partner Brian J. Malkin Speaks on Biologics Center Breakout at FDLI's Annual Meeting in Washington, D.C.

On April 23, FLH Partner Brian J. Malkin will speak at FDLI's Annual Meeting in Washington, D.C. in the Concurrent Breakout Session for the Center for Biologics Evaluation and Research ("CBER"). During the Concurrent Breakout Session, representatives from each FDA center will discuss the three most important developments from last year and their three most important goals in 2013. Following the FDA center representative presentations, food and drug law stakeholders responding to their presentations. This year Diane Maloney, Esq., Associate Director for Policy, CBER, will be presenting for CBER, and Mr. Malkin will be responding as a food and drug law stakeholder. Scott Cunningham, Partner, Covington and Burling, will moderate the session. The Concurrent Breakout Sessions are always a big draw for attendees at FDLI's Annual Conference, and Mr. Malkin hopes to see you there and at the CBER Breakout Session.

April 19, 2013

FLH Partner Brian J. Malkin Speaks at FDLI's Annual Conference on CBER Breakout Panel Next Week

FDLILogo.jpgNext week, FLH Partner Brian J. Malkin will speak at Center for Biologics Evaluation and Research ("CBER") Panel at FDLI's Annual Meeting held in Washington, D.C. on April 23-24. The CBER Panel will be held on the first day of the Conference, Tuesday, April 23, from 2-3:30 p.m. FDLI has posted the key two presentations by CBER Representatives Diane Maloney, Esq., Associate Director for Policy, and Mary Malarkey, Director, Office of Compliance and Biologics Quality. The panel will be moderated by Scott Cunningham, Partner, Covington & Burling LLP, and also includes Michael S. Reilly, Executive Director, Alliance for Safe Biologic Medicines, and Mark S. Robbins, Ph.D., Vice President, Clinical Regulatory Affairs, DiaMedica USA, Inc.

As demonstrated by the presentations already posted by Maloney and Malarkey, the panel has a lot of ground to cover, and we as panel members have been providing feedback for additional topics to address as time permits. All of us hope for a lively discussion following the initial presentations and hope to see you there!

FDLI's Annual Conference is the largest (750+ attendees expected) and longest running conference for the FDA-regulated industry, which now includes tobacco products. FDLI's program brings together experts from the Federal Government, industry, private sector bar, consulting organizations, and academia in one place to discuss legal, regulatory, policy, and economic issues falling in FDA's vast authority. FDA Commissioner, Margaret A. Hamburg, M.D., will provide the Keynote Address the first day, with other plenary sessions featuring FDA's top regulatory and compliance officials, breakouts for all of FDA's centers and a sessions featuring compliance and liability issues. The next day features top cases in food and drug law, concurrent breakouts in emerging issues, an address from FDA's Chief Counsel, Elizabeth Dickinson, and plenary sessions in interagency dynamics and the role of the media in food and drug law, regulation, and policy.

April 12, 2013

Biosimilars and "Pay-for-Delay" Settlements on the Table in White House's Proposed Budget

Obamabudget.jpgOver the past months, there has been a lot of speculation (see recent blogs here , here, and here) whether the White House's proposed budget would cause a sequester situation for FDA, resulting in potential layoffs or program cuts, in an era of new user fees for generic drugs and biosimilar biological products. While initial reports and temporary budget fixes (called continuing resolutions) appeared to keep FDA's user fees intact and available for use, FDA's Commissioner, Margaret A. Hamburg, M.D., recently reported to members of a biotechnology trade association, the Massachusetts Biotechnology Council ("MassBio"), that it was not clear what would happen with user fees in the new federal budget.

Released on April 10, the White House's proposed fiscal year 2014 budget is a mixed bag that has been called a "political document rather than a serious piece of legislation" with a "series of bargaining positions" that "would bleed pharma." On the one hand, the plan would appear to confirm that FDA's user fees would not be sequestered, given that it supported the $4.7 billion in total program budget requested by FDA, which included user fees that would help fund over 90 percent of the requested increases. On the other hand, the budget includes a myriad of proposals that would change the way the government pays for medical care and products. For example, Medicare (senior citizens' drug coverage) Part D manufacturer discounts for branded drugs would be increased from 50% to 75% in 2015 (rather than 2020) and low-income individuals would be pushed more to generic drugs by increasing certain copayments for branded drugs and lowering certain copayments for generic drugs.

Many of the more controversial proposals were nestled in a document called "Reducing the Deficit in a Smart and Balanced Way". Here, the White House proposes, among other things, several items to purportedly lower drug costs, including: 1) authorizing the Federal Trade Commission to stop companies from entering into certain "pay-for-delay" agreements (see below) and 2) beginning in 2014, to reduce biologic product exclusivity from 12 years to 7 years and prohibit additional periods of exclusivity for minor changes to product formulations. These two items could open up some unanticipated debate regarding the White House's budget.

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April 3, 2013

UK's NICE To Have the Crucial Role in the Value-Based Pricing of Medicines from 2014

Bristish Notes.pngThe United Kingdom's ("UK's") method of controlling the prices its Department of Health pays for innovative medicines has been, up until now, the Pharmaceutical Price Regulation Scheme ("PPRS"). The PPRS has been a voluntary scheme run over many years between the Department of Health and the branded pharmaceutical industry represented by the Association of the British Pharmaceutical Industry ("ABPI"). Its objective was to deliver the provision of safe and effective medicines at reasonable prices to the National Health Service ("NHS") while promoting innovation, the rapid uptake of new clinically and cost effective medicines, and in a sustainable manner.

Over recent years, the National Institute for Health and Care Excellence ("NICE") has been taking on a more visible and key role. Its job has been to improve outcomes for people using the NHS by producing evidence-based guidance by way of advice to healthcare professionals and to develop quality standards and measures for those providing health care services and also providing information to practitioners and managers in both health and social care.

The UK Government's response to the Health Select Committee report of 2012-2013 on NICE was to re-establish NICE as a new statutory body giving it a key position in the healthcare system. It gives NICE the role of deciding the value-based pricing of medicines. Thus, value-based pricing will replace the current PPRS, when it expires in January 2014.

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April 1, 2013

Biosimilar Meeting Guidance Issued by FDA

dna.jpgOn April 1, FDA issued a Federal Register Notice announcing a new draft biosimilars guidance, "Formal Meetings Between the FDA and Biosimilar Biological Product Sponsors or Applicants". This is the latest in FDA's new biosimilar guidances for 2013, which FDA has announced in earlier meetings this year would be coming to help spur the filing of a biosimilars application, which FDA has called 351(k) applications based on the section in the Public Health Service Act ("PHS Act"). As of a few weeks ago at the Massachusetts Biotechnology Association's ("MassBio's") Annual Meeting, which we blogged on here, FDA's Commissioner, Margaret A. Hamburg, M.D., continued to report that FDA has not received a single 351(k) application to date.

The Guidance focuses on formal meetings for 351(k) applications and the associated requirements or performance goals from the Biosimilar User Fee Act of 2012 ("BsUFA"), which was enacted as part of the Food and Drug Administration Safety and Innovation Act ("FDASIA"). In particular, the Guidance discusses the principles of good meeting management practices ("GMMPs") and describes standardized procedures for requesting, preparing, scheduling, conducting, and documenting such formal meetings.

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March 19, 2013

MassBio Features Hamburg Keynote at Annual Meeting - Part 2 of 2

monoclonal antibodies.pngOn March 14-15, the Massachusetts Biotechnology Council ("MassBio") held its Annual Meeting in Cambridge, Massachusetts. The Meeting also featured a Keynote from FDA Commissioner Margaret A. Hamburg, M.D. (see related blog here). Key themes at the Meeting were the importance of the Cambridge/Boston biotechnology community for advancing new therapies and the unique resources available in the area that have made it an industry leader. Some of the Cambridge/Boston advantages discussed were the intellectual research capital (local universities such as Harvard and Massachusetts Institute of Technology), venture capital, and local biotechnology businesses, such as Biogen Idec and Genzyme, as well as other biotechnology companies that now have offices in the Cambridge/Boston area and are seeking partnerships to develop new products, such as AstraZeneca, Pfizer, Merck, Novo Nordisk, and Sanofi.

On the second day, Hamburg described here "special affection" for the Cambridge/Boston region dating back to her days at Harvard, saying that she hopes D.C. "would be as efficient and congenial as here." Hamburg said that the Cambridge/Boston region is a life sciences enterprise fueled by top notch research and medical care with the top five NIH-funded hospitals and a "biotech supercluster second to none" with "a remarkable 500 biotech and pharma companies here, and some thirty venture capital firms."

Hamburg described FDA as striving for true collaboration and regulatory flexibility with industry, including MassBio, and has been hearing that industry wants more clarity, certainty, transparency with decisions. Hamburg said that FDA is trying to have creative approaches--not a one size-fits-all approach. To this end, Hamburg described approaches that FDA has taken with four new products from the Massachusetts area: 1) Inclusig® for two rare forms of leukemia, 2) Juxtapid® (an orphan drug), 3) Linzess® for irritable bowl syndrome, and 4) Kalydeco® for cystic fibrosis. In addition, Hamburg highlighted new provisions in the Food and Drug Administration Safety and Innovation Act ("FDASIA") for expedited approvals, citing 31 breakthrough therapy designation requests, of which 9 have been granted, 10 denied, 11 pending, and 1 withdrawn. To help with more companies taking advantage of this new process, FDA will be publishing a new guidance shortly, Hamburg announced.

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March 18, 2013

MassBio Features Biosimilars and Crowley Keynote at Annual Meeting - Part 1 of 2

humanonachip.jpgOn March 14-15, the Massachusetts Biotechnology Council ("MassBio") held its Annual Meeting in Cambridge, Massachusetts. The Meeting featured key topics such as biosimilars and a Keynote from John Crowley, Chairman and CEO of Amicus Therapeutics.

On the first day of the conference, Crowley exemplified many of the speakers' entry in biotechnology, which originated with a family member or friend with a disease requiring development of a biotechnology product. For Crowley, it was his two children Megan and Patrick, were diagnosed with a severe neuromuscular disorder, Glycogen storage disease type II, known as Pompe's disease. Rather that sitting still to wait for a cure, Crowley became involved in the process, first moving to Princeton, New Jersey, to be close to doctors specializing in the disease and leaving his job with Bristol-Myers Squibb. He later took a position as CEO of Novazyme Pharmaceuticals, a biotechnology research company located in Oklahoma City founded by Dr. William Canfield, which was conducting research on a new experimental treatment for the disease. Novazyme was acquired by Genzyme Corporation, which was then the world's third largest biotechnology company. Crowley was put in charge of Genzyme's global Pompe program, becoming the largest research and development effort in the company's history.

Through these efforts, an experimental enzyme replacement therapy was developed, and Megan and Patrick Crowley received the therapy, which Crowley credits with saving his children's lives. Crowley went on to become President and CEO of Orexigen Therapeutics and was named the President and CEO of Amicus Therapeutics, based in Cranbury, New Jersey, which he helped take public in 2007. Crowley's efforts were documented in a Wall Street Journal article and other publications, which ultimately resulted in Harrison Ford working to bring the story to life in a major motion picture, Extraordinary Measures.

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