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May 2, 2013

MassBio Sustainability Forum Offers Insights for the Biotechnology Community

Sustainability.pngOn May 1, the Massachusetts Biotechnology Council included a Forum on sustainability. "Addressing the Evolving Sustainability Demands in the Biotech Industry". Sustainability has become a hot topic for many companies including pharmaceutical and other manufacturers of FDA-regulated products because of the need to provide for long-term maintenance of our earth's resources. Sustainability broadly is the capacity to endure. In ecology the word describes how biological systems remain diverse and productive over time. Long-lived and healthy wetlands and forests are examples of sustainable biological systems. For humans, sustainability is the potential for long-term maintenance of well being, which has ecological, economic, political, and cultural dimensions.

The Forum featured a mix of industry speakers: Moderator, Wayne Bates, Ph.D., P.E., Vice President and Sustainability Director at Capaccio Environmental Engineering, Inc. and panelists:
James McCabe, Sustainability Manager, Global Operations/Services, Waters Corporation; Tony Meenaghan, Senior Director of Facilities Management, Environmental Health and Safety and Engineering, EMD Serono, Inc.; David Reilly, Global Energy Manager, Novartis; Timothy Stoll, LEED, Director for Development and Leasing, BioMed Realty Trust.

The speakers explained that sustainability programs have arisen due to the concern about how companies impact in environment. The goal is that a sustainability program "meets the needs of the present generation without compromising the ability of future generations to meet their needs." Sustainability is not meant to be trendy, it is about environmental stewardship, social well being, and economic prosperity over long term (bearable, viable, equitable). In process, many of these programs have been random acts of sustainability rather than a coordinated plan.

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July 20, 2012

Europe Reviews Gene Therapy Favorably for the First Time

genetherapy.jpgThe promise of gene therapy as a commercially-available tool for treating genetic disorders may finally be inching closer to reality. On July 20, 2012 the European Medicines Agency ("EMA") Committee for Medicinal Products for Human Use ("CHMP") took the first step towards making gene therapy treatment a reality by recommending the gene therapy, Glybera® (alipogene tiparvovec), for marketing approval in the European Union ("EU"). Glybera®, which is indicated for the treatment of lipoprotein lipase ("LPL") deficiency, was developed by Netherlands-based, uniQure.

Patients lacking LPL enzymes are unable to break down fats. The CHMP press release states that:

So far, management of patients with the disorder consists of strict reduction of dietary fat to less than 20% of the daily caloric intake. It is very difficult to comply with such a dietary regimen and as a consequence many patients experience life-threatening pancreatitis attacks requiring admission to hospital.
Due to the difficult circumstances faced by individuals suffering from LPL deficiencies that trigger severe or multiple pancreatitis attacks, the CHMP has deemed the treatment benefits afforded by this gene therapy to be worth its risks.

Fundamentally, gene therapy is a method by which exogenous "functioning" or "normal" DNA replaces "abnormal" DNA that is responsible for a particular genetic disorder. Gene therapy was originally touted in the 1970s as a potential way to treat genetic disorders.

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July 17, 2012

FLH Partner Brian J. Malkin Speaks at ACI's Clinical Trials Conference on July 17, 2012 in Boston

Frommer Lawrence & Haug LLP Partner Brian J. Malkin will present a controversial topic concerning the type of clinical trials that FDA will require for biosimilar product applications entitled: "Safely Conducting Biosimilars Clinical Trials: Understanding FDA's Requirements for Biosimilar Clinical Trials". Mr. Malkin will address what FDA has said about its "selective and targeted approach" for biosimilar clinical trials and how biosimilar applicants may be able to rely on certain clinical trial data from the innovator's product, as well as assessing the risks and benefits for conducting such clinical trials. Mr. Malkin's presentation is part of the American Conference Institute's 14th Advanced Summit on Clinical Trials: Ensuring Safe and Compliant Domestic and International Clinical Trials on July 17-18, 2012 in Boston.

July 6, 2012

Nanotechnology: User Fee Bill Increases Funding for FDA Studies

nanotechnology.bmpThe Food and Drug Administration Safety and Innovation Act, otherwise known as the User Fee Bill, has passed through Congress and awaits the President's signature. This Bill, mostly known for implementing user fees for generic drug applications, also provides new programs to foster the study of nanomaterials in products regulated by FDA. Section 1126 of Title XI, Subsection C calls for the Secretary to "intensify and expand activities related to enhancing scientific knowledge regarding nanomaterials, ... to address issues relevant to the regulation of those products, including potential toxicology, the potential benefit of new therapies derived from nanotechnology, the effects of nanomaterials on biological systems, and the interaction of such nanomaterials with biological systems." This provision mirrors the Nanotechnology Regulatory Science Act of 2011 introduced by Sens. Ben Cardin (D-MD) and Mark Pryor (D-AR) last year, which stalled after its introduction in the Senate.

Nanomaterials utilize nanotechnology--manipulation of matter on the atomic and molecular scale. Nanomaterials, measured in billionths of a meter, range from 1- 100 nanometers (nm) and are used in a range of products, from paint and sunscreen to drugs and cosmetics. In 2010, the National Science Foundation estimated that nanotechnology-based products and manufacturing would add 2 million jobs and $1 trillion dollars in revenue to the world economy by 2015. These nanoproducts have different physical, chemical, and biological properties than conventionally-scaled materials, and some speculate that these properties may involve unknown risks to humans and our environment.

Written by Caroline Bercier

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The importance of nanomaterials has been recognized for some time. President Clinton advocated nanotechnology research, and President George W. Bush increased funding for nanotechnology development in 2003 with the passage of The 21st Century Nanotechnology Research and Development Act. However, this will be the first time that Congress has mandated that FDA study nanotechnology to evaluate the safety and toxicity of nanomaterials in consumer goods and products.

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June 7, 2012

SoloHealth's Station Kiosk Approved: Awareness Drives Action with New Self-Service Solution to Healthcare

On June 5, SoloHealth announced that FDA approved its next-generation SoloHealth Station, a self-service health care device which strives to assist people with both education and awareness about their health. SoloHealth, an Atlanta-based company, has worked to empower consumers through the use of technology to achieve a healthier, and consequently better, America. With the recent FDA approval and an aggressive rollout plan for 2012, the SoloHealth Station is taking the lead in using self-service technology to develop a healthier America.

Just prior to receiving the inaugural Intel Innovation Award, CEO and Founder of SoloHealth, Bart Foster, described the SoloHealth Station as a kiosk equipped with both the "functionality and technology to drive consumer education, awareness, and ultimately action." SoloHealth makes clear that the kiosk will not replace an examination by a medical professional. Essentially, the SoloHealth Station will provide consumers with an initial health screening and encourage them to visit a health care provider for a follow-up examination.

Written by Elizabeth Zinke

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The SoloHealth Station offers a highly personalized, targeted, and interactive opportunity for consumers by employing a touch user interface and interactive video to guide consumers through the simple health tests. It will allow consumers to screen their vision, blood pressure, weight, and body mass index in less than seven minutes. In addition, the SoloHealth Station will provide a symptom checker as well as an overall health assessment. These services are provided free of charge.

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May 24, 2012

First Stem-Cell Therapy Receives Approval in Canada

Stem Cell.jpgOn May 17, Health Canada approved Prochymal® (remestemcel-L) for the treatment of acute graft-versus-host disease ("GvHD") in children. GvHD, a complication of bone marrow transplantation, occurs when the white blood cells in the grafted tissue recognize the host's organs as foreign and attack the host. Acute GvHD usually presents within three months of the bone marrow transplant, and most often causes damage to the liver, skin, stomach, and intestines. Acute GvHD is categorized as stage I, II, III, or IV based on the number of affected areas, and the severity of the damage. Prochymal® is the first approved treatment for GvHD worldwide, and it receives eight and one half years of exclusivity in Canada.

Currently, the standard treatment for GvHD is intravenous steroids, which suppress the immune system to prevent attack of the transplant recipient's organs. Treatment with steroids, however, is only successful in 30 to 50 percent of patients, as suppressing the immune system can lead to infections or even death. Health Canada approved Prochymal® for use in children with acute GvHD where steroid treatment has been unsuccessful.

Prochymal® is comprised of mesenchymal stem cells from the bone marrow of a healthy adult donor. Prochymal®'s approval falls under Canada's Notice of Compliance with Conditions ("NOC/c") Pathway, which requires that Osiris Therapeutics, the drug's maker, continue with confirmatory clinical testing. Osiris presented data to Health Canada showing clinically meaningful responses in 61 to 64 percent of children with severe GvHD that failed to respond to steroids. This data is very encouraging, considering that severe GvHD has a mortality rate of close to 80 percent. Health Canada relied on this data in granting regulatory approval to Osiris. Prochymal® is currently available in the U.S. (and several other countries) under the Expanded Access Program, for patients "2 months to 17 years with acute GvHD, grades B-D, that is not responsive to steroid therapy."

FDA has required additional data from Osiris before it will consider Prochymal® for approval, so Osiris plans to apply for approval in the U.S. later this year after it collects the required information. Osiris also plans to use Prochymal® for the treatment of Crohn's disease, heart attack, COPD, Type I diabetes, and for potential Acute Radiation Syndrome. The company is currently conducting clinical studies for these indications.

May 21, 2012

Biotechnology-Derived Pharmaceuticals Guidance Issued

DNA.jpgOn May 17, FDA's Centers for Drug Evaluation and Research and Biologics Evaluation and Research released an Addendum to Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals. Recognizing the need for greater harmony in the regulatory standards for biotechnology-derived pharmaceuticals among Europe, Japan, and the United States, FDA issued the original Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals Guidancein July 1997.

According to the original guidance, preclinical safety evaluations have three goals: (1) to identify an initial safe dose and subsequent dose escalation scheme in humans; (2) to identify potential target organs for toxicity and for the study of whether such toxicity is reversible; and (3) to identify safety parameters for clinical monitoring. That guidance set forth a basic framework for the preclinical safety evaluation of biotechnology-derived pharmaceuticals to "improve the quality and consistency of the preclinical safety data supporting the development of biopharmaceuticals."

The Addendum is meant to "complement, provide clarification on, and update" the original guidance. According to FDA, following the Addendum's guidance should "facilitate the timely conduct of clinical trials, reduce the use of animals . . . and reduce the use of other drug development resources." Specifically, the Addendum addresses the following topics: species selection, study design, immunogenicity, reproductive and developmental toxicity, and assessment of carcinogenic potential.

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February 16, 2012

New Pharmacogenetics Guidelines Adopted by European Medicines Agency

by Howard E. Rosenberg, Ph.D.

gene.jpgLate last year, the European Medicines Agency ("EMA") published a new guideline, "Guideline on the use of pharmacogenetic methodologies in the pharmacokinetic evaluation of medicinal products" to provide a framework for where it is recommended that pharmacogenetics should be implemented in the drug development process. At the same time, the guideline recognizes that pharmacogenetics may not be equally important for every drug.

Patients are not all identical and different individuals may well react to a particular medicine in diverse ways. For example the manner in which a patient absorbs and/or metabolizes a particular drug may well differ form one to another. In recent years there has been a rapid development in the understanding of the influence of genes on interindividual differences in drug action. Hence the pharmacokinetics of many medicinal products is prone to interindividual variability, caused by several factors such as gender, age, weight, impaired renal and hepatic function, and genetics.

In the field of pharmacogenetics, interindividual variability in genes influencing or predicting the outcome of drug treatment (e.g., genes encoding drug transporters, drug metabolizing enzymes, drug targets, biomarker genes) is studied in relation to efficacy of drug treatment and adverse drug reactions. A knowledge of genetic factors influencing absorption, distribution, metabolism and excretion ("ADME") is centered on drug metabolism. Genetic variations in metabolizing enzymes may lead to: (i) increased or decreased clearance of the parent drug or pharmacologically active or toxic metabolites, (ii) increased or decreased production of active metabolites of the respective prodrugs, or (iii) increased or decreased formation of toxic products.

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December 16, 2011

EU Ban on Stem Cell Patents Spurs Debate

by Howard Rosenberg

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The EU ban on stem cell patents, issued in an October 2011 Court of Justice of the European Union ("CJEU") opinion, continues to generate controversy and input from a wide variety of viewpoints. This week Aurora Plomer, professor of law and bioethics at Sheffield University argued that the ban not only imperiled Europe's competitiveness but also the protection of human rights.

The professor attacked the decision from a legal perspective arguing the ruling suffered from several flaws. She stated that the drafting history of the EU directive on biotechnological inventions, showed that the intention was never to render unpatentable lawful research and that there isn't a uniform pan-European view on what respect for human dignity means with regard to human embryos, contrary to the position taken by the Court of Justice. She also pointed out that the ruling was inconsistent with the settled law of the European Court of Human Rights in that that court has applied a "margin of appreciation" doctrine in relation to the rights of the embryo accepting that the status of the embryo can differ from state to state.

The CJEU's decision, binding on all member states, was that the concept of a human embryo had to be understood in a very wide sense ensuring respect for human dignity and, consequently, stem cell lines whose derivation required the destruction of human embryos were not patentable under European law. However, if the European Convention on Human Rights has no requirement for a consistent application or understanding of embryonic human rights amongst the EU member states then the CJEU's decision may well be at odds with it. According to Professor Plomer,. the CJEU ruling raises important questions about the compatibility of EU law with the European Convention on Human Rights, which the EU is bound to respect.

Professor Plomer also felt that the ban would deter investment and frustrate the EU's goal of harmonising laws to speed up the development of regenerative treatments that are anticipated to offer huge therapeutic potential, e.g. in Alzheimer's and Parkinson's disease.

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November 17, 2011

HEMACORD Gets Thumbs-Up from FDA, Provides Needed Access for Blood Banks

by Fitz Beckwith Collings

Buddy_Blood_Drop.jpgOn November 10, FDA approved HEMACORD, a cord blood biologic, for use in patients with "disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment." The active ingredient in HEMACORD consists of hematopoietic progenitor cells that express the cell surface marker CD34. The potency of cord blood is determined by measuring the number of total nucleated cells ("TNC") and CD34+ cells, and cell viability. Each unit of HEMACORD contains a minimum of 5 x 10(8) total nucleated cells with at least 1.25 x 10(6) viable CD34+ cells at the time of cryopreservation.

Cells expressing CD34 (CD34+ cell) are normally found in the umbilical cord and bone marrow as hematopoietic cells, a subset of mesenchymal stem cells, endothelial progenitor cells, endothelial cells of blood vessels but not lymphatics (except pleural lymphatics), mast cells, a sub-population dendritic cells (which are factor XIIIa negative) in the interstitium and around the adnexa of dermis of skin, as well as cells in soft tissue tumors. Matching for at least 4 of 6 HLA-A antigents, HLA-B antigens, and HLA-DRBI alleles is recommended. The HLA typing and nucleated cell count for each individual unit of HEMACORD are documented on the container label or accompanying records.

Cord blood may used to treat disorders that generally result in diminished or lost capacity to form new blood cells, e.g., following radiation therapy. The undifferentiated hematopoietic cells in cord blood migrate to the bone marrow once inside the body. Once there, they mature and divide, replenishing lost or absent blood cell populations and function, including immune function, of blood-borne cells of marrow origin. In patients with enzymatic abnormalities due to certain severe types of storage disorders, mature leukocytes resulting from HPC-C transplantation may be able to circulate and improve cellular functions of some native tissues. HEMACORD is the first hematopoietic progenitor cell-cord cell therapy licensed by the FDA.

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November 14, 2011

Health Information Technology--Who Should Regulate?


EMR.jpgOn November 8, the Institute of Medicine ("IOM") released a report, "Health IT and Patient Safety: Building Safer Systems for Better Care". The IOM Committee had been charged with summarizing existing knowledge of the effects of health information technology ("IT") on patient safety, making recommendations to the Department of Health and Human Services ("HHS") regarding specific actions federal agencies should take to maximize the safety of health IT-assisted care, and making recommendations concerning how private actors can promote the safety of health IT-assisted care, and how the federal government can assist private actors in this regard.

The IOM Committee concluded that current market forces are not adequately addressing the risks associated with the use of health IT. No federal agency is currently charged with monitoring the safety of health IT, i.e., the software, hardware, and systems that record and manage patients' health information. The Report recommended that HHS should publish an action and surveillance plan within twelve months that includes a schedule for working with the private sector to asses the impact of health IT on patient safety and minimizing the risk of its implementation and use. The Report also recommended that HHS fund a new Health IT Safety Council, which would operate within an existing voluntary consensus standards organization, to evaluate criteria for assessing and monitoring the safe use of health IT to enhance safety. All health IT vendors should be required to publicly register and list their products with the Office of National Coordinator for Health IT ("ONC"), the Report continues, and HHS should establish a mechanism for reporting health IT-related deaths, serious injuries, or unsafe conditions.

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November 2, 2011

Opioid Painkiller Overdose Epidemic--Need for Effective REMS Evident


morphinepills.jpgOn November 1, the Centers for Disease Control and Prevention ("CDC") published in its publication, Morbidity and Mortality Weekly Report ("MMWR"), the results of a study that confirmed the epidemic of overdoses of opioid pain relievers ("OPRs") has continued to worsen particularly from 1999-2008. OPR deaths now account for more drug overdose deaths than heroin or cocaine combined and represent nearly 75% of all prescription drug overdose deaths. Drug overdose deaths (36,450) are now almost as high as the number of deaths from motor vehicles (39,973), which is the leading cause of injury death in the United States.

What is also alarming is that the OPR overdose rates have continued to rise despite increased warnings and recommendations over the past decade for voluntary education of health-care providers about more cautious use of OPRs. According to the MMWR, the death rate of drug overdoses of 11.8 per 100,000 population (nearly 100 people per day) is about three times the rate in 1991. Since 1999, most of the drug overdose increases have been attributed to prescription drug overdoses. In 2009 alone there were 1.2 million emergency department visits related to misuse of pharmaceuticals, which represented a 98.4% increase from 2004, compared to 1.0 million for use of the illegal drugs heroin and cocaine.

According to the MMWR, sales of OPRs quadrupled between 1999 and 2010 so that by 2010 enough OPRs were sold to mediate every American adult with a typical dose of hydrocodone every 4 hours for 1 month. Increased use of OPRs has contributed to overall increases in overdose death and nonmedical use. Yet the total number of prescribers responsible for the increase appears to be small: one study found only 3% of physicians accounted for 62% of the OPRs prescribed. Once prescribed, OPRs may be diverted for nonmedical uses (i.e., to patients for whom the drug was not prescribed or for other uses not attributed to pain for whom the drug was prescribed) or sold on the street. In 2010, 4.8% of the U.S. population 12 and older reportedly used OPRs nonmedically. This, in turn, cost insurance companies up to $72.5 billion in health-care costs.

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November 1, 2011

Vaccine Immunization Exemptions May Be Putting Children at Risk--What Should FDA Do?


childimmunization.jpegOn October 31, a preliminary report of a new California study was presented at the American Public Health Association in Washington, D.C. that appears to demonstrate that state vaccination "personal belief exemptions" are resulting in "hot spot" zones where children are at greater risk of exposure to preventable infectious diseases such as measles, mumps, and rubella.

The new California study analyzed state health department statistics for personal belief exemptions among kindergartners. Personal belief exemptions are affidavits signed by parents indicating that "all or some immunizations are contrary to my beliefs," which permits children to avoid otherwise mandated vaccines. Twenty states allow personal belief exemptions including California.

According to the study, in 2010 for every 100 children in a California kindergarten, 2.3 had skipped immunizations because of one or more personal belief exemptions. Interestingly, the exempted children tended to cluster in certain schools, where an average of 16 out of 100 of their peers also claimed exemptions. Some schools had one in five kindergartners with parental exemptions for vaccinations. More than 7,000 kindergartners across California attended these schools, including 2,700 who were exempted.

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October 31, 2011

Drospirenone's Possible Increased Risk of Blood Clots

by Julie E. Kurzrok

birthcontrolpills.jpegIt has long been known that there is an increased risk of cardiovascular complications including, for example, blood clots known as venous thromboembolisms ("VTEs") that result from the use of combined hormonal contraceptives ("CHCs"). Most CHCs are birth control pills containing the hormones estrogen and progestin. Recently, several new CHCs have been introduced into the market, and concerns have arisen regarding whether the cardiovascular risks are greater among these new preparations. The new CHCs are drospirenone/ethinyl estradiol tablets, the norelgestromin/ethinyl estradiol transdermal patch, and the etonogestrel/ethinyl estradiol vaginal ring. Drospirenone is a synthetic progestin, and the drospirenone tablets are marketed under the names Yaz®, Yasmin®, Beyaz®, and Safyral® (along with various generics). On October 27, 2011, the FDA published the final study report from its evaluation of these risks along with a safety statement regarding the possible increased risk of VTEs from drospirenone tablets.

FDA's study was a retrospective study entitled, "Combined Hormonal Contraceptives (CHCs) and the Risk of Cardiovascular Disease Endpoints" and it compared the risk of blood clots, among other things, in women who took three newly-marketed CHCs to women who took four older CHCs. The study examined the medical histories of 835,826 women from 2001 to 2007 and found that women taking the newly-marketed CHCs, including the drospirenone tablets, had a significantly higher risk of VTEs. Among only the users of the new formulations, the drospirenone tablets were the only ones with a significantly higher risk of VTEs. In conclusion, FDA stated that this study provided further evidence to link the drospirenone tablets with an increase in VTEs as compared to the other low-dose CHCs.

However, FDA has also seen conflicting results from six other studies of drospirenone pills and the risk of VTEs. Because of the conflicting results, FDA will hold a joint meeting with scientific advisors from the Reproductive Health Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee on December 8, 2011. At the meeting FDA will review the results of its study and discuss the risks and benefits of drospirenone birth control pills, especially the risk of blood clots associated with these pills.

September 27, 2011

Stem Cell Ruling Appealed . . . Again

by Erin Lawrence

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On September 19, 2011, Plaintiffs James Sherley and Theresa Deisher, filed a notice of appeal in their lawsuit Sherley v. Sebelius. The appeal asks that the Court of Appeals for the District of Columbia reverse the District Judge Royce Lamberth's July 27th decision that the government can continue to finance embryonic stem cell research. (See our previous blog here).

Judge Lamberth dismissed the Plaintiffs' lawsuit in July citing the Court of Appeals April holding that government-backed research on embryonic stem cells is likely lawful under the Dickey-Wicker Amendment. The appeal now seeks an opposite decision from the same Court of Appeals. The appeal requests that the Court of Appeals block the U.S. Health and Human Services Department and the National Institute of Health from spending federal funds on research involving human embryonic stem cells.

The scientist hired a Christian law firm, Jubilee Campaign to file the appeal. The Jubilee Campaign is spondering the "Law of Life Project" which is dedicated to "defending the right to life and dignity of the human being from biological conception. . . ."

The next step will be for Court of Appeals to set a schedule for submission of legal briefs and oral arguments. Although Plaintiffs' success in the appeal may be a far shot--especially given that the Court of Appeals has already decided that it is highly likely that government funding of embryonic stem cell research is legal--a victory for Plaintiffs' would put the stem cell battle on a path to the U.S. Supreme Court.