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Morgen is het bal

72 Posts
Pagina: «« 1 2 3 4 »» | Laatste | Omlaag ↓
  3. [verwijderd] 29 juli 2012 13:34
    quote:

    Antoni schreef op 29 juli 2012 13:13:

    Ik neem aan dat je het echt wilt weten. Ze worden aangeboden tussen de €0,32 en €10,00. Er is op aantal aanbiedingen geboden tussen €0,32 en €0,42. Het loopt nog niet storm.
    Ik snap dat mensen graag een leuk dealtje slaan, maar na vermarkting in 2013/ 2014 en of eventuele partnerschappen sta je er veel leuker voor, om nog maar te zwijgen over de mogelijkheden voor de mensen met een extra lange adem vwbt het hemofilie B project; natuurlijk zou het goed kunnen dat een grote jongen voordat hier echt baanbrekende ontwikkelingen in zijn de boel al heeft overgenomen; maar dan zal de prijs toch goed zijn, omdat het potentieele rekensommetje heel mooi zal uitvallen..
  5. flosz 3 augustus 2012 17:51
    Rare Diseases: 5 Recent Reasons to Cheer

    #1: GENE THERAPY PENDING APPROVAL
    #2: FINDING HOMES FOR STALLED DRUGS
    #3: SPEEDING FDA APPROVAL
    #4: EASING INSURANCE ACCESS
    #5: IDENTIFYING DISEASE GENES

    blogs.scientificamerican.com/guest-bl...
    ….
    “The triglycerides dropped, but after 60 days they trended back up. The primary endpoint had failed, but the secondary endpoint was recurring episodes of pancreatitis – and they found a statistically significant, or close to it, decrease,” explained James Wilson, MD, PhD, editor-in-chief of Human Gene Therapy and professor of pathology and laboratory medicine at the University of Pennsylvania, who developed the vector. Tracking a few more patients, work not yet published, may have led the EMA’s Committee for Medicinal Products for Human Use to finally recommend approval, after three rejections.
    …………..
  6. flosz 4 augustus 2012 09:47
    Inside story of how uniQure turned around EMA on Glybera gene therapy

    Persistence pays off

    By Stephen Hansen
    Senior Writer

    After two and a half years and four tries, uniQure B.V.’s gene therapy Glybera alipogene tiparvovec finally won the backing of the European Medicines Agency’s CHMP. Approval by the European Commission would make Glybera the first gene therapy to win approval outside China, and complete the story of management’s campaign to remind policy makers about Europe’s commitment to rare diseases.
    The tale also provides a window into the inner workings of EMA and its scientific committees.
    Along the way, the process cost uniQure’s publicly traded predecessor
    nearly all its institutional investors and ultimately forced Amsterdam Molecular Therapeutics Holding N.V. to go private.
    However, with each review, Glybera got a little closer to acceptance. According to CEO Joern Aldag, the first two reviews saw the committees accept that Glybera was safe. It took the third and fourth reviews to convince CHMP that Glybera provides a clinical benefit.
    On July 20, CHMP recommended approval of Glybera under exceptional circumstances to treat lipoprotein lipase (LPL) deficiency, a rare autosomal recessive disorder affecting about 1 in 1 million people. The disease is characterized by severe hypertriglyceridemia leading to acute pancreatitis.
    The EC’s decision is expected within three months. If Glybera’s crosses the goal line, Aldag says the review will have done more than
    validate the company’s adeno-associated virus (AAV) vector technology, but also have provided EMA with the experience to better assess gene therapies that have yet to come before it.

    General to particular
    AMT submitted the MAA in January 2010. According to Aldag, what followed
    was more than a year of answering questions from CHMP and the Committee for Advanced Therapies (CAT). CAT and CHMP are independent EMA
    committees. They separately review and vote on an MAA for an advanced therapy, which is defined as a gene therapy, cell therapy or tissue engineering product. When an MAA involves a gene or cell therapy, a rapporteur and co-rapporteur are chosen from CAT to prepare the initial
    assessment report.
    The EC established CAT in 2007 to help foster innovation for advanced therapies. CAT has 31 members including one from each EU member state and four appointed by the EC — two to represent clinicians and two to represent patient associations. CHMP includes 32 members, including one appointed by each member state and five members chosen from among experts nominated by member states or EMA.
    CHMP must have 17 positive votes for the committee to reach a positive opinion on an MAA.
    During the first part of an MAA review for an advanced therapy, CAT and CHMP send questions to the company. After the assessment report has been created and prior to the CHMP vote, an oral Q&A session may be held between CAT, CHMP and the company. Initial questions about Glybera from
    CAT and CHMP showed the committees were wrestling with gene therapy overall. “There were a lot of questions from CAT and CHMP about gene therapy in general. They were not directly linked to Glybera, but were going way beyond,” Aldag said.
    Aldag said it was clear CHMP was being very cautious despite data, which he said showed a good safety profile for Glybera (see “Glybera’s Clinical Program”). CHMP’s first opinion in June 2011 was negative. Aldag said the assessments of both CAT rapporteurs — who were from the U.K. and Germany — were negative. He said both the CAT and CHMP votes
    also “were clearly negative.” Aldag did not recall the actual votes of
    the CHMP, and the committee could not provide them to BioCentury.
    The day of the decision, AMT’s stock fell 67% to €0.64, leaving the company with a market cap of €15 million ($21.4 million).
    The company requested a re-examination after being invited to do so by CHMP. Under the re-examination process, CAT replaces the rapporteurs, but the new reviewers examine the same data set. Aldag noted this process gives a company another shot at presenting its case. “When you submit the original dossier, you answer lots of questions in a piecemeal fashion. When you do a re-examination, you are allowed to rewrite the answers in a more coherent way,” he said. “We learned what they were asking for from the questions,” he said. Thus, the second time around, “we could put the
    answers into a different context and explain them differently.”
    The reformatted answers seemed to help. Both rapporteurs, this time from
    Italy and Latvia, recommended approval, and CAT recommended approval of
    Glybera under exceptional circumstances. Approval under exceptional circumstances requires companies to conduct additional postmarketing studies to confirm the clinical benefit. But CHMP did not follow suit. While the committee no longer regarded safety as an issue, according to Aldag, it had concerns regarding efficacy. According to CHMP’s summary, “The applicant had not provided sufficient evidence of a persistence of effect in lowering blood fats in a clinically relevant manner and there were too few patients for whom sufficiently long-term data were available.” According to Aldag, the median follow-up among 27 patients in the clinical program supporting the MAA was almost two and a half years at the time of submission. The follow-up data ranged from 1.4-
    3.6 years. He said the CHMP vote was negative by a slight margin. On Oct. 21, 2011, the day of the announcement, AMT’s stock dropped 51% to €0.38, giving the company a market cap of €8.9 million ($12.4 million).
    As a result, Aldag said almost all of the company’s institutional investors dumped the stock. In their place, day traders began speculating on the shares. Chairman Sander Slootweg told BioCentury the new shareholder base made it “more difficult to raise money and expect the company’s value to increase upon meaningful news flow.” Slootweg is also
    co-founder and managing partner of Forbion Capital.

    AMT decided to go private. In February, it announced that privately held
    uniQure would acquire all the company’s assets and certain liabilities. Long-term AMT investor Forbion put €6 million ($8 million) into the newco.
    While Slootweg wouldn’t go so far as to say taking AMT public was a mistake, he did note that, in hindsight, being a public company saddled AMT with additional burdens like investor relations that a private
    company would not encounter. On the other hand, Slootweg noted, it would have been unlikely that AMT could have raised as much money from private
    investors in 2007 as it did in its €50 million ($66.9 million) IPO that year.

  7. flosz 4 augustus 2012 09:48
    Orphan networking
    By this time, Aldag and other members of management had begun reaching out to members of the EC and the European Parliament seeking to discuss the commission’s objectives for fostering innovation and getting new therapies to patients.
    Rare diseases have been a priority in recent EU legislation and EC directives. The EC’s 2007 health strategy highlighted Orphan diseases as a priority, while the Sixth and Seventh Framework Programmes for R&D cited rare diseases as a research area of focus. In 2008, the EC’s communication to the European Parliament and council urged support for developing rare disease networks across the EU and improving access to Orphan drugs. Aldag said several individuals in the EC and Parliament, whom he did not identify, told both him and CHMP that the committee’s procedures were getting in the way of innovation. These individuals,
    along with the company, were frustrated that CAT and its rapporteurs found the data acceptable for approval, but CHMP did not, he said.
    “What was happening may be correct in the sense of the formal rules, but it was not correct in the sense of what the EC is trying to accomplish” regarding innovation, Aldag said.
    uniQure also petitioned the Directorate General for Health & Consumers (DG SANCO), the EC committee that ratifies CHMP decisions. At DG SANCO’s meeting in January this year, rather than simply ratify the CHMP recommendation, the committee asked CHMP to re-examine Glybera for an indication restricted to LPL-deficient patients who have experienced either severe or multiple pancreatitis attacks.
    The company agreed to the restricted label with CHMP, and the MAA moved to
    a third review. Because the MAA had not been withdrawn, additional data could not formally be added to the application. However, the company was able to supply long-term data in response to questions from CAT and CHMP.
    As it pursued this new regulatory tack, the company had been conducting additional analyses of the 27 patients treated with Glybera. Specifically, the company gathered complete histories of each patient, and each episode of pancreatitis was adjudicated by independent experts in a blinded fashion. The additional analysis showed Glybera significantly reduced the frequency of pancreatitis attacks compared to the period before treatment. The third review of the Glybera MAA
    came in April this year. Aldag said CAT again recommended approval, and once again the application failed to win CHMP’s backing.
    EMA spokesperson Monika Benstetter told BioCentury that while CHMP informally consulted CAT on its opinion, the advanced therapy panel did not provide a formal assessment report of Glybera for the restricted indication. Although CHMP voted 16-15 in favor of Glybera, the application failed to muster the 17 votes needed for a majority, according to Aldag. One member missed the vote due to illness. The restricted indication limited the data set to 12 patients. In its decision, CHMP said efficacy data from the small number of patients “was not sufficiently convincing,” and that the reduced risk of pancreatitis could have been due to other factors. Part of the issue was that CHMP thought data from the Phase II/III trial showing a reduction in postprandial plasma chylomicrons was the most convincing, but these data were available for only five patients. Chylomicrons are lipoprotein particles consisting mostly of triglycerides that circulate in the plasma.
    CAT had recommended that additional data on chylomicron clearance could be
    collected in the postmarket setting, but CHMP did not agree.
    Recognizing “the difficulty of obtaining data in this rare disease,” CHMP did not request any additional data but instead invited uniQure to request a re-examination — a fourth review — during the committee’s June meeting. CHMP did not give a reason for the invitation. Time may have been on Glybera’s side in finally winning CHMP’s backing, as the
    company continued to accumulate data for the therapy. Again, the company
    couldn’t formally submit the data, but it could provide anecdotal updates on the long-term data.

    The CHMP vote in the re-examination on July 19 was not disclosed. In a statement, acting Chair Tomas Salmonson said: “Additional analyses by CAT have added to the robustness of the data provided and allowed CHMP to conclude that the benefits of Glybera are greater than its known risks.”
    According to Benstetter, the “additional analyses” consisted of a formal assessment report of Glybera for the subpopulation of severe LPL deficient patients. Specifics of the analyses will not be disclosed until after the EC decision is announced. If approved, uniQure will be required to build a registry of LPLdeficient patients treated with Glybera. Data collected from patients will include long-term measures of chylomicron clearance.

    Trendsetter?
    “Going through this process was very important,” Aldag said. “The traditional way of thinking is based on large, randomized controlled trials, not adapting them, not changing endpoints — all these things are practically impossible in ultra-Orphan drug development.”
    “If you are trying to push Orphan drug development and promote advanced therapeutics, you have to change the way you are looking at these things,” he added. “The positive thing is that we’ve been through a process at the end of which the highest levels of the European Commission, the CHMP and CAT all now understand that the way you have to look at Orphan drugs is different.”
    He said that experience will benefit uniQure’s other gene therapy programs, as well as other companies developing advanced therapeutics or Orphan drugs in Europe. Aldag said a decision has not yet been made on uniQure’s strategy for Glybera in the U.S. The company plans to hold a pre-IND meeting with FDA in the next few months, after which he said uniQure will have a better understanding of whether additional clinical trials will be required and whether the company will seek a development or marketing partner in the region.

    UniQure has five other gene therapy programs, the most advanced of which is AMT-060. The AAV vector encoding the Factor IX gene has completed a Phase I/II trial to treat hemophilia B. Aldag suggested the experience also may have triggered EMA to improve the coordination of its committees when conducting evaluations. In April, only five days after CHMP announced the third negative opinion for Glybera, EMA Executive Director Guido Rasi announced the formation of the Scientific Coordination Board.
    The board brings together the heads of EMA’s seven scientific committees, including CAT and CHMP. The goal is to improve communication between the committees so that standards for development of medicines are consistent throughout a product’s lifecycle.
    Aldag said the board is meant to discuss how EMA’s scientific evaluation “has to change going forward to improve the number of drugs being approved and to make the process more efficient and strategically sensible.”

    www.biocentury.com/biotech-pharma-new...

  8. flosz 4 augustus 2012 09:48
    Glybera’s clinical program
    Lipoprotein lipase (LPL) deficiency is characterized by severe hypertriglyceridemia leading to acute pancreatitis. uniQure B.V.’s gene therapy Glybera alipogene tiparvovec is designed to replace the absent enzyme and thus restore proper clearance of triglycerides from plasma. The product comprises an AAV vector encoding the LPL gene for one-time
    delivery by intramuscular injection. According to CEO Joern Aldag, of the roughly 10,000 people with LPL deficiency worldwide, only 2,000-5,000 may be eligible for clinical trials because many are either undiagnosed or the patient’s disease is not severe enough to be eligible. In the Netherlands, the company’s home base, 19 people have been diagnosed with LPL deficiency. Glybera’s clinical program included three interventional trials in a total of 27 patients. In two of the studies, totaling 22 patients, the primary endpoint was reduction in fasting plasma triglycerides. An additional post hoc analysis of the first 22 patients showed a significant reduction in the incidence of acute pancreatitis.
    The third trial, a pivotal Phase II/III trial in five patients, had a primary endpoint of a reduction in postprandial plasma chylomicrons.
    Normal LPL enzymes pull chylomicrons out of circulation to be metabolized in the muscle. In LPL-deficient patients, the lipoprotein particles continue to circulate and grow in number and size, eventually clogging up the pancreas and causing pancreatitis. According to Forbion Capital General Partner Sander van Deventer, clearance or reduction in chylomicrons is a clear signal of LPL enzyme functionality. van Deventer,
    formerly CSO and CEO of Amsterdam Molecular Therapeutics Holding N.V., is a cofounder and board member of uniQure.
    In the pivotal Phase II/III, the data showed that a single treatment with Glybera resulted in a significant reduction in the number of chylomicrons produced after a meal at 14 and 52 weeks after treatment. Aldag said an 80-90% reduction in chylomicrons was seen in patients compared to levels prior to treatment.
    — Stephen Hansen
  16. flosz 9 oktober 2012 08:51
    Endgame: Glybera Finally Recommended for Approval as the First Gene Therapy Drug in the European Union
    Seppo Ylä-Herttuala
    In an Editorial published earlier this year in Molecular Therapy,1 I described the regulatory uncertainty surrounding the evaluation of Glybera (alipogene tiparvovec) by the European Medicines Agency (EMA). Glybera is an adeno-associated viral vector engineered to express lipoprotein lipase in the muscle for the treatment of lipoprotein lipase deficiency. As with a typical TV serial, its regulatory saga has now had four acts, leading recently to a final recommendation by the EMA to approve Glybera in the European Union.2 Although this is excellent news for the gene and cell therapy communities and for companies working in this area, the regulatory process that led to this positive outcome is still not without problems.
    Amsterdam Molecular Therapeutics submitted its original application to the EMA in January 2010. The specialty Committee of Advanced Therapies (CAT), established by the EMA to review gene and cell therapy products, recommended against a marketing authorization for Glybera, and this view was consequently adopted by the Committee on Human Medicinal Products (CHMP), which makes final recommendations for marketing authorization in the EU. Amsterdam Molecular Therapeutics responded by requesting reexamination of the application, which led to the CAT’s reversing its earlier ruling and recommending approval of the drug. It was therefore a great surprise when, in October 2011, the CHMP once again rejected marketing authorization based on its view that “the dossier had not provided sufficient evidence of a persistence of effect in lowering blood fats in a clinically relevant manner and there were too few patients from whom sufficiently long-term data were available.” Indeed, lipoprotein lipase deficiency is an ultra-orphan disease affecting only one in a million people. According to publicly available information, the clinical results were based on only 27 patients. Following the negative decision, Amsterdam Molecular Therapeutics faced severe financial problems, and a new private company (UniQure) was established with rights to Amsterdam Molecular Therapeutics’ assets.
    Apparently after intensive lobbying and some political pressure, in January 2012 the European Commission, which usually ratifies CHMP recommendations, unexpectedly asked the committee to reexamine Glybera for an indication restricted to lipoprotein lipase–deficient patients who have experienced either severe or multiple pancreatitis attacks, which are serious problems in these patients. Further analysis showed that patients receiving Glybera experienced significantly fewer pancreatitis attacks than they had before treatment. The third review resulted in a CHMP vote of 16 to 15 in favor of marketing authorization—1 vote shy of the absolute majority (in this case 17) necessary for a positive opinion. Thus, the third round of review produced a negative opinion, even though a narrow majority on the CHMP was in favor of approval. Evaluation of Glybera for the restricted indication had limited the clinical material to 12 patients, and the CHMP was concerned that the data showing a reduction in postprandial plasma chylomicrons was apparently derived from only 5 patients. Recognizing “the difficulty of obtaining data in this rare disease,” the CHMP invited UniQure to request reexamination for a fourth time, which resulted in the CHMP’s handing down a majority vote on 19 July in favor of authorization. Apparently, the CHMP concluded that the benefits of Glybera in this subset of patients were greater than the known risks. This positive decision should be ratified by the European Commission later this year.
    This lengthy and tortuous approval process raises several questions. Frequent reapplications are very time-consuming and expensive, which in this case led to the demise of Amsterdam Molecular Therapeutics, although thankfully private investors were able to continue the process. Nevertheless, investors will shy away from supporting biotech companies unless greater clarity and predictability can be achieved in the process of regulatory evaluation and approval. The Glybera saga also highlights problems specific to ultra-orphan drugs—because obtaining large-scale phase III data with a very limited number of patients is virtually impossible, procedures to handle these indications must be further developed.
    Reapplications are not entirely uncommon during the regulatory evaluation of traditional small-molecule drugs and monoclonal antibodies. However, it is noteworthy that the first three products that have been evaluated by the CAT and the CHMP have required reapplications and reexaminations. Among these products, a chondrocyte cell therapy product was eventually approved, whereas an adenoviral thymidine kinase gene therapy for malignant glioma was not. Clearly, researchers and investors developing gene and cell therapies must be prepared for a prolonged and iterative evaluation process. Despite these problems and difficulties, the final opinion in favor of Glybera is encouraging news for the gene and cell therapy communities, and hopefully evaluation of the twists and turns of the plot of this saga will help to streamline the regulatory processes of other gene and cell therapy products so that this new area of medicine can eventually fulfill its promise in human medicine.
    www.nature.com/mt/journal/v20/n10/pdf...

    Gene therapies need new development models
    www.nature.com/polopoly_fs/1.11521!/m...

    Dutch Life Sciences & Health Conference December 11
    Trends and Developments in Dutch Life Sciences:
    H. (Hans) Preusting PhD, MBA
    uniQure B.V.
    Vice President Business Development
    Speaker workshop Market authorisation: successes and failures
    Hans Preusting joined uniQure’s predecessor company AMT in August 2006 and is currently responsible for Business Development.
    www.dutchlshconference.com/index.php/...
  18. flosz 12 oktober 2012 16:01
    Labyrint radio, d.d. 07-10-2012:
    Gentherapie op de markt
    Er wordt al heel lang veel verwacht van de mogelijkheden van gentherapie. Maar ondanks die verwachtingen is er nog geen getherapeutisch middel op de markt. Eén dezer dagen is het dan toch zover: het middel Glybera, waarmee een zeldzame stofwisselingsziekte te behandelen is, mag in Europa verkocht worden. Professor Frank Staal is in de studio om te vertellen over de moeite die het heeft gekost om zover te komen. (+Sander van Deventer)
    www.wetenschap24.nl/programmas/labyri...

    Over Frank Staal: www.lumc.nl/0000/12296/hoo/1102100307...
72 Posts
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