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You have full access to this article via your institution. Download PDF EU AGENCY MAKES FIRST RECOMMENDATION FOR ADVANCED THERAPY The European Medicines Agency (EMEA) has recommended that

the European Commission approves a cell-based therapy called ChondroCelect, developed by TiGenix. The lowdown: In December 2008, a new regulation on advanced therapy medicinal products

(ATMPs) came into effect in the European Union (EU) (regulation no. 1394/2007). The regulation established the Committee for Advanced Therapies (CAT), which now assesses all marketing

authorization applications for ATMPs developed in the EU and reports its findings to the Committee for Medicinal Products for Human Use (CHMP). The first ATMP, which was authorized for

marketing by the EMEA's CAT and CHMP on 26 June 2009, is ChondroCelect (consisting of characterized viable autologous cartilage-forming cells, expanded _ex vivo_, expressing specific

marker proteins) for the treatment of adults with single-symptom cartilage defects of the femoral condyle of the knee (International Cartilage Repair Society grade III or IV). The

cartilage-forming cells (chondrocytes) are taken from a small biopsy of healthy cartilage from the patient, grown outside the body and then surgically re-implanted with the aim of repairing

cartilage defects by the formation of durable cartilage. The ultimate goal of the treatment is to reduce the risk of developing knee osteoarthritis in the long term. Also, the CAT has

adopted its first scientific recommendation on the classification of an ATMP for a somatic-cell therapy medicinal product intended for the treatment of chronic venous leg ulcers. It is

composed of substantially modified human allogeneic fibroblasts and keratinocytes, administered in conjunction with fibrin as a structural component. All requests for classification are

assessed by the CAT (see http://www.emea.europa.eu/htms/human/advanced_therapies/atmp_classification.htm). NEW ION CHANNEL DATABASE AVAILABLE ONLINE The International Union of Basic and

Clinical Pharmacology (IUPHAR) has announced the publication of an ion channels database that includes both voltage-gated ion channels (VGICs) and ligand-gated ion channels (LGICs). THE

LOWDOWN: The IUPHAR database (http://www.iuphar-db.org/) contains peer-reviewed information about pharmacological, chemical, genetic, functional and pathophysiological properties of human,

rat and mouse genes. The current release adds detailed information on 8 VGIC and 3 LGIC families (specifically, 141 VGIC and 71 LGIC subunits), to the information on the 354 non-sensory G

protein-coupled receptors (GPCRs), including orphan receptors, that has been available since 2005. Curation of information about the five remaining ion channel gene families is ongoing.

ALNYLAM EXPANDS PATENT POOL FOR NEGLECTED DISEASES Alnylam Pharmaceuticals has donated over 1,500 patents (issued or pending) on its RNA interference technology to the patent pool for

neglected tropical diseases (NTDs), which was established by GlaxoSmithKline in March this year. THE LOWDOWN: By donating these patents, the aim is provide the knowledge needed for the

development of new medicines for the treatment of US FDA-defined NTDs (tuberculosis, malaria, blinding trachoma, Buruli ulcer, cholera, dengue and dengue haemorrhagic fever, racunculiasis,

fascioliasis, human African trypanosomiasis, leishmaniasis, leprosy, lymphatic filariasis, onchocerciasis, schistosomiasis, soil-transmitted helminthiasis and yaws). In addition to its

intellectual property, Alnylam will also provide technical know-how on a royalty-free, non-profit basis in the least developed countries through licensing agreements with qualified third

parties.  Image courtesy of F. Collins, Centers for Disease Control, Atlanta, Georgia, USA. Merck has also recently announced a collaboration with the not-for-profit Drugs for Neglected

Diseases initiative (DNDi) to discover and develop improved treatments for NTDs. Through the collaboration, Merck will contribute small-molecule assets and related intellectual property

through a non-exclusive, royalty-free licence to DNDi to conduct early development programmes of drug candidates. The Drug Discovery Unit at the University of Dundee has also recently

announced a collaboration with the DNDi. The Unit at Dundee will develop a dedicated leishmaniasis drug discovery group that will form consortia with academic centres such as the Structural

Genomics Consortium in Oxford, UK, and the London School of Hygiene and Tropical Medicine, UK. FDA ALLOWS FURTHER DEVELOPMENT OF OXFORD BIOMEDICA'S CANCER VACCINE Following a review by

the US FDA of Oxford Biomedica's Phase III trial of TroVax in renal cancer, the company can continue to develop the product for various metastatic cancer indications. THE LOWDOWN:

TroVax is a gene-based tumour vaccine that uses a modified poxvirus vector to deliver the tumour antigen gene _5T4_ and induce an immune response against the 5T4 antigen. Oxford Biomedica

started a Phase III clinical trial of TroVax in patients with advanced or metastatic renal cell carcinoma in November 2006 (called TRIST (TroVax Renal Immunotherapy Survival Trial)).

However, in July 2008, a preliminary analysis by an independent data safety monitoring board indicated that the trial would not meet its primary end point. The board recommended that the

trial continue, without further vaccinations, to allow patient follow-up. Oxford Biomedica have now announced that the TRIST trial did show a survival advantage in certain subsets of

patients with renal cancer and the FDA has invited them to submit adaptive Phase II–III trial designs in metastatic colorectal cancer. JAPAN APPROVES FIRST FOLLOW-ON BIOLOGIC The Japanese

regulatory authorities have approved their first biosimilar product — Sandoz's version of recombinant human growth hormone somatropin (Omnitrope). THE LOWDOWN: On 25 June 2009, Sandoz

announced that their follow-on version of somatropin has been approved for use in Japan for the treatment of children with growth hormone deficiency, as well as growth disturbance associated

with Turner's syndrome or chronic renal insufficiency. Earlier this year, the Japanese Ministry of Health, Labour and Welfare (MHLW) published guidelines for a national biosimilar

regulatory pathway based on similar scientific principles to the approval pathway of the European Medicines Agency (EMEA). The MHLW are not the only regulators that could benefit from the

EMEA's experience in biosimilars. It was reported from the annual Drug Information Association meeting that the US FDA will learn from the EMEA's experience in biosimilars when

defining their own regulatory pathway, according to Steven Kozlowski, director of the FDA's Office of Biotechnology Products. This comes at a time when the details of a regulatory

pathway for such products are under considerable debate in the United States. The main point of contention is the length of data exclusivity before another company can make a regulatory

filing for a follow-on product that uses data generated by the innovator company for the original product. The Senate Health, Education, Labor and Pensions Committee has voted 16–7 in

support of an amendment to the health-care reform package that provides 12 years of data exclusivity for innovative biologics. This is in addition to growing support in the House for H.R.

1548 (The Pathway to Biosimilars Act), which also recommends at least 12 years of exclusivity and has 133 co-sponsors (at the time of going to press). A competing bill, H.R. 1427 (The

Promoting Innovation and Access to Life-Saving Medicine Act), which recommends 5 years of data exclusivity, has 14 co-sponsors (at the time of going to press). ALLIANCE FORMED TO ADVANCE

CELL-BASED THERAPIES An Alliance for Regenerative Medicine has been formed between universities, life sciences companies and health-care investors, to be based in Washington, DC, USA. THE

LOWDOWN: The Alliance for Regenerative Medicine aims to promote regulatory, research and reimbursement policies that will foster innovation in regenerative medicine (such as stem cell-based

therapies). Also, the Alliance will serve as a source of information about regenerative medicine for policymakers, the media and the general public. The advocacy organization has a number of

initial charter members: the universities that will participate include the Georgia Institute of Technology, Stanford University, the University of Washington and Wake Forest Institute for

Regenerative Medicine; the life science companies include Aldagen, Fate Therapeutics, Geron, iZumi, Johnson & Johnson and Maxcyte; the investor organizations include Caufield and Byers,

Kleiner, Perkins and Proteus Ventures. In addition, the Genetics Policy Institute is a charter member. It was formed in 2003 with the express purpose of promoting and defending stem cell

research and its application in medicine, by leading a global network of stakeholder groups representing patient advocates, scientists, physicians and health-care professionals,

industrialists, bioethicists, lawyers, educators and policymakers. At the time of going to press, the full list of charter members is expected to be published by the end of July 2009. RIGHTS

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