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Orphan drugs treat rare diseases affecting less than 200,000 people in the United States. The manufacturing process for orphan drugs is generally complex and time-consuming, making them susceptible to manufacturing quality issues and shortages. Dm g shortages of any kind are detrimental to patient health and safety. But orphan drug shortages are especially concerning because orphan drugs treat rare diseases with no other alternative treatments, and one drug maker typically produces the entire supply. The market exclusivity that an orphan drug maker experiences also compounds orphan drug shortages by preventing competitive products in the market. Therefore, when orphan drug shortages occur due to manufacturing interruptions or discontinuances, patients may be left without an available treatment. Recently, courts have held that drug manufacturers do not have a duty to keep manufacturing drugs and supply them to the market. Therefore, patients do not have tort remedies available when there is a disruption in drug supply. Unfortunately, there is not a silver bullet to solve orphan drug shortages, but there are various solutions to mitigate them. This Note focuses specifically on Congress and the FDA, and how both institutions should not only aim to prevent orphan drug shortages, but find alternative, compensatory solutions for patients harmed by these shortages. This Note will explain orphan drug shortages, government efforts to resolve them, discuss Genzyme's recent orphan drug shortage as an example of why there needs to be action, and the negative consequences of failing to act. This Note will then propose various solutions that provide a two-pronged approach of both preventive and compensatory solutions that best serve patients harmed by orphan drug shortages. One such preventative measure is that the FDA should require orphan drug makers to include back-up manufacturing requirements as a part of their New Drug Application or Biological License Application filings. Another solution is for the FDA to consider the establishment of a stockpile repository for orphan drugs. Congress should also pass legislation to allow the FDA to establish a compensation fund that would provide monetary relief to patients harmed by orphan drug shortages. Ultimately, there needs to be more action by Congress and the FDA so that patients in need of orphan drugs or harmed by orphan drug shortages will no longer feel betrayed and "orphaned" by drug companies and the government. [ABSTRACT FROM AUTHOR]

The article offers gene therapy news briefs as of March 2014. The companies uniQure BV, and Celladon Corp. raised 135.8 million dollars through initial public offerings. Researchers from University of Oxford, and Imperial College London conducted the first clinical trial of a retinal gene therapy for choroideremia. Alnylam Pharmaceuticals Inc., and Genzyme Corp. formed an alliance worth 1 billion dollar which included equity, and research and development funding.

The article discusses the results of the journal's 2011 Careers Top Employers Survey and examines the hiring practices and research of biotechnology and pharmaceutical companies which were well rated including Vertex Pharmaceuticals Incorporated, Genzyme Corp., and Novo Nordisk. Science and medical research company executives including Andy Chan of Genentech, Bryan Iams of Bayer, and Susan Bunz of Pioneer Hi-Bred, a DuPont business discuss how they recruit scientists, the corporate culture of their institution, and the effect of the economy on their institutions.

The article discusses the growing participation of patients to clinical research. It was observed that patients look for faster drug development, better recruitment for clinical research, and actions from regulatory settings. Highlighted are the cases of biotechnology firm Genzyme on the exclusive license of patent for Fabrazyme and of John and Aileen Crowley who raised funds for nonprofits supporting research into their children's Pompe disease. However, there is a concern on conflicts of interest for the relationships of advocates and the industry.

Abstract This paper examines the problem of risk mitigation in virtual organizations (VOs). We begin by discussing risk propensity in virtual organizations, and draw on a variety of research to [...]

Plerixafor [Mozobil™, AMD 3100, JM 3100, SDZ SJD 791] is a bicyclam derivative that acts as a stem cell mobiliser by blocking the CXCR4 chemokine receptor. Plerixafor was synthesised by Johnson Matthey (AnorMED) in collaboration with the Rega Institute of Leuven, Belgium. Plerixafor is in phase III clinical trials in stem cell transplantation among cancer patients. Plerixafor blocks CXCR4, which triggers the rapid movement of stem cells out of the bone marrow and into circulating blood. These cells can then be collected and used in stem cell transplant procedures. Plerixafor had been available for partnering in Europe. However, decisions concerning partnering arrangements were deferred by AnorMED until top-line clinical data became available (expected in 2007). In November 2006, Genzyme Corporation completed its acquisition of AnorMED. Genzyme intends to commercialise plerixafor in >50 countries throughout the world using its existing transplant business. Evotec OAI was selected by AnorMED to support it in the chemical development of plerixafor. Evotec OAT will use EVOdevelop™, its integrated chemical and pharmaceutical development platform, to complete the full validation of the process to plerixafor, including process research and development, cGMP manufacturing and analytical work. Evotec OAI will also be responsible for producing the relevant Chemical Manufacturing Control (CMC) documentation for regulatory filings. Top line results from the phase III studies are expected in the second quarter of 2007 and, assuming these are successful, the marketing submissions are planned for the US in 2007 (launch in 2008), and for Canada and Europe in 2008. Plerixafor has orphan drug status for stem cell transplantation in cancer patients in the US and the EU. AnorMED (now Genzyme) decided to pursue a full Marketing Authorisation Application (MAA) in Europe for plerixafor in stem cell transplant. Previously, the company had been planning on filing a CMA (Conditional Marketing Authorisation) in this region. The change in strategy requires additional phase II trials in the five major EU markets. Multicentre phase II trials with plerixafor have begun in Canada and Germany in approximately 50 patients with non-Hodgkin's lymphoma and multiple myeloma (studies EU2I and C201). Enrolment has been completed in a US-based, multicentre, phase II trial (study 2105) of plerixafor plus G-CSF in patients with multiple myeloma and non-Hodgkin's lymphoma. This study is designed to optimise the administration schedule of this combination therapy regimen. Plerixafor has completed a phase II study (study 2104) in multiple myeloma and NHL patients in combination with chemotherapy. A US-based phase II pilot study (study 2108) with plerixafor as a single mobilising agent in multiple myeloma patients undergoing stem cell transplant is underway. Another US-based phase II pilot study (study 2106) is evaluating plerixafor in combination with the standard mobilisation regimen, G-CSF, in patients with Hodgkin's disease undergoing stem cell transplant. AnorMED completed a phase II study (study 2101) evaluating the potential of plerixafor in combination with G-CSF as a therapy for stem cell transplantation compared to G-CSF therapy alone. The study involved patients with multiple myeloma and patients with NHL. Results indicated that the combination regimen was significantly superior to G-CSF treatment alone in stem cell mobilisation. Further trials are planned for plerixafor, to expand its use in transplant and in other indications including one to investigate the potential of plerixafor to improve the effectiveness of chemotherapy in patients with leukaemia. Phase I trials have been completed. [ABSTRACT FROM AUTHOR]

Presents legal and regulatory updates on biotechnology and pharmaceutical industries as of September 2003. Fine imposed by the British Office of Fair Trading to Genzyme Ltd. regarding the supply of drugs for the treatment of Gaucher disease; Need to revise the regulations that limit the number and location of community pharmacies in Great Britain; Overview of the intention to make the definition of generic products more precise.

Provides information on α-galactosidase, an enzyme replacement therapy for the treatment of Fabry's disease developed by Genzyme General. Launch of Fabrazyme in the European Union in August 2001; Rights of Genzyme General to a broad patent covering the expression of α-galactosidase from Mount Sinai School of Medicine; Drug development history.

The article presents a list of studies about wet age-related macular degeneration (AMD) including "Safety and Tolerability of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AMD)," by Genzyme, "Study of Intravitreal Microplasm in Relieving Vitreo-Macular Adhesion in Neovascular Age-Related Macular Degeneration (AMD)," by the University of California-Los Angeles/ThomboGenics, and "Reduced Fluence PDT With Visudyne in Combination With Lucentis for Age-Related Macular Degeneration," by the Oklahoma State University Center for Health Sciences/Novartis.

The article reports on the signing of a $1.9 billion agreement between Genzyme and Isis Pharmaceuticals in the U.S. According to the author, the agreement is to in-license the cholesterol-lowering antisense drug mipomersen. He added that Genzyme will pay $325 million up front for the powerful drug mipomersen, an antisense treatment that slashes cholesterol levels. Moreover, mipomersen is a second-generation antisense phosphorothioate oligonucleotide inhibitor that is thought to act by binding mRNA encoding apolipoprotein B-100.

Finding solutions for patients with unmet needs is at the heart of Genzyme's innovative medical research. While small molecules and enzyme-replacement therapies have made significant strides in improving patients' quality of life and expectancy, the solutions to many conditions, such as heart disease or CNS diseases, rely on the capacity of the body to regenerate cells. Cell and gene therapy lend themselves well to providing solutions for these illnesses with no current cure, by harnessing the body's natural ability to heal. Promising research in these fields continues to evolve and constitutes a long-term investment in addressing serious unmet medical needs in various therapeutic areas. Over the past 15 years, Genzyme has established itself as a pioneer in shaping the research and commercial application of cell and gene therapy. Genzyme's first commercially available innovative cell therapies -- Carticel® and Epicel® -- have provided a solid foundation to move into new, cutting-edge areas. [ABSTRACT FROM AUTHOR]

The article reports on three initiatives that address the reputation of the health care industry in the European Union. First, the first worldwide clinical trials portal is launched to provide doctors, patients and their families with simple access to the information on ongoing and completed clinical trials of drugs and vaccines. Second, a Healthcare Manifesto is released by EuropaBio aimed at the well-being of the innovative biotechnology industry and of the patients. Lastly, the U.S. biotechnology firm Genzyme Corp. significantly expanded its engagement in Europe by establishing a new discovery research facility in Cambridge, England.

Presents news briefs related to pharmaceutical industry in the U.S. as of September 2003. Financial performance of Amgen Pharmaceuticals in second quarter of 2003; Acquisition of SangStat Medical Corp. by pharmaceutical company Genzyme Corp.; Information about follow-on offerings in biotechnology sector in the month of July.

1. Introduction Glucocerebrosidase (GCR; EC 3.2.1.45) is a membrane-associated lysosomal enzyme [1] responsible for the hydrolysis of glucocerebroside to glucose and ceramide [2, 3]. Deficiency in the GCR activity results [...], Impaired activity of the lysosomal enzyme glucocerebrosidase (GCR) results in the inherited metabolic disorder known as Gaucher disease. Current treatment consists of enzyme replacement therapy by administration of exogenous GCR. Although effective, it is exceptionally expensive, and patients worldwide have a limited access to this medicine. In Brazil, the public healthcare system provides the drug free of charge for all Gaucher's patients, which reaches the order of $ 84 million per year. However, the production of GCR by public institutions in Brazil would reduce significantly the therapy costs. Here, we describe a robust protocol for the generation of a cell line producing recombinant human GCR. The protein was expressed in CHO-DXB11 ([dhfr.sup.-])cells afterstable transfection and gene amplification with methotrexate. As expected, glycosylated GCR was detected by immunoblotting assay both as cell-associated (~64 and 59kDa) and secreted (63-69kDa) form. Analysis of subclones allowed the selection of stable CHO cells producing a secreted functional enzyme, with a calculated productivity of 5.14pg/cell/day for the highest producer. Although being laborious, traditional methods of screening high-producing recombinant cells may represent a valuable alternative to generate expensive biopharmaceuticals in countries with limited resources.

Five hypothetical mergers could change the industry and provide a model for its future. [ABSTRACT FROM AUTHOR]