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With the approval by the European Commission in November 2012.....
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Most expensive medicine: Glybera breaks Guinness world record Amsterdam, The Netherlands -- Glybera, developed by private Dutch firm uniQure, the first gene therapy to win approval in the West, is expected to arrive soon with a price tag of more than a million dollars, setting the new world record for the Most expensive medicine, according to the World Record Academy: www.worldrecordacademy.com Glybera is the first gene therapy approval in an independently regulated healthcare market. Professor John Kastelein, of the University of Amsterdam, said the therapy would have a 'dramatic impact' on patients. Glybera is expected to arrive soon with a price tag of more than a million dollars, eclipsing the likes of Alexion's ($ALXN) Soliris, which costs $440,000 a year.www.worldrecordacademy.com/medical/mo...
Jörn Aldag zieht aus dem Hin und Her einen allgemeinen Schluss. Es gebe rund 5000 seltene Erkrankungen, die ähnlich wie der LPL-Mangel auf ein einziges defektes Gen zurückzuführen und deshalb für die Wissenschaft besonders zugänglich seien. Die Aufsichtsbehörden hätten es in der Hand, die Entwicklungskosten und damit auch die später geforderten Erstattungen für neue Behandlungsmethoden für solche Krankheiten sinken zu lassen, indem sie den Herstellern in diesen Fällen weniger Aufwand für die klinischen Studien aufbürdeten. Ook goed nieuws voor de mensen met deze ziekten.
Ontwikkelingen gaan heel snel. Steeds nieuwe technieken.
kijk eens welk clubje hier beschreven wordt tussen alle grote jongens als Genzyme/sanofi en GSKseekingalpha.com/article/1136641-gene...
En uniQure heeft een vacature uitstaan voor een junior onderzoeker die zich zal bezig houden met gentherapie voor Huntington’s Disease. Over 4 jaar kunnen we de resultaten verwachten.
Prof. Dollar schreef:
En uniQure heeft een vacature uitstaan voor een junior onderzoeker die zich zal bezig houden met gentherapie voor Huntington’s Disease. Over 4 jaar kunnen we de resultaten verwachten.
Misschien komt een van de 'groten' wel op het idee van een overname.
en nog een van fierce biotechwww.fiercebiotech.com/story/biotech-v... let op deze uitspraak: "Hemophilia B is the next priority," says Astley-Sparke, with treatments for other conditions like porphyria to follow. His responsibilities as U.S. president also include scouting for fresh capital, which may involve a possible IPO at some point as uniQure builds up its commercial arm.
In een eerder persbericht (van toen nog AMT) staat: "Hemophilia B is a rare disease, occurring in 1 in 30,000 people, almost always in males. The total number of patients in Europe and the USA together is estimated to be between 35,000 and 40,000". Het is geen aantal dat uniQure een blockbuster maakt. Zal dat ooit beuren?
Prof. Dollar schreef:
In een eerder persbericht (van toen nog AMT) staat:
"Hemophilia B is a rare disease, occurring in 1 in 30,000 people, almost always in males. The total number of patients in Europe and the USA together is estimated to be between 35,000 and 40,000". Het is geen aantal dat uniQure een blockbuster maakt. Zal dat ooit beuren?
Beste zou zijn als de techniek zo baanbrekend is dat een grote overneemt. Anders is het weer een kwestie van 10 jaar met het risico dat er vernieuwende technieken beschikbaar komen.
Ik snap het allemaal niet. Hoezo geen blockbuster als je 40.000 patienten hebt. Als 25% daarvaan het gebruikt a 100.000 euro per jaar heb je 1 miljard omzet. 25% a 25.000 euro per jaar is 250 Miljoen omzet. Leuke cashflows hoor. Dan nog iets wat ik in het Engels hierover op een ander forum heb geschreven: I cannot believe the remarks I read about the price of Glybera and people doubting the market access side of it. You just pay 100.000 dollar per symptom free good quality life year for let's say 10 years. That adds up to: 1.000.000 dollars. This is now common practice in orphan drug land. So why would a therapy like Glybera not be worth this?
Het moet allemaal nog geconcretiseerd worden. Geduld is echt een schone zaak in deze. Ooit worden de volhouders beloond. Het kan alleen nog 5-10 jaar duren. Mijn inziens is er geen project waarbij je een mooier rendement kan maken dan bij UniQure; als aandeelhouder zijnde..
Prof. Dollar schreef op 30 januari 2013 20:29 :
In een eerder persbericht (van toen nog AMT) staat:
"Hemophilia B is a rare disease, occurring in 1 in 30,000 people, almost always in males. The total number of patients in Europe and the USA together is estimated to be between 35,000 and 40,000". Het is geen aantal dat uniQure een blockbuster maakt. Zal dat ooit beuren?
Hemofiliepatienten kosten in hun patientenleven lang plm 10-12mln aan behandelingen...als die kosten met 75% van 10 mln worden teruggebracht en bij 50% vd 30.000 patienten werkzaam is, dan gaat het alsnog om een omzet van 37,5 miljard.... Dát vind ik best een blockbuster...
Houdt er wel rekening mee dat dit eenmalige omzet is. De patient heeft slechts 1 behandeling nodig en is dan klaar. Stel dat alle 30.000 patienten het eerste jaar worden behandeld a EUR 100.000 per behandeling, dan behaalt Uniqure in dat jaar een omzet van EUR 3 mld en in de jaren daarna niets meer.
Niks is wat het lijkt schreef:
Het moet allemaal nog geconcretiseerd worden. Geduld is echt een schone zaak in deze. Ooit worden de volhouders beloond. Het kan alleen nog 5-10 jaar duren. Mijn inziens is er geen project waarbij je een mooier rendement kan maken dan bij UniQure; als aandeelhouder zijnde..
Een tijdspanne van 10 jaar is veel te lang. De ontwikkelingen gaan zo snel, dat alles wat nu nieuw is dan al lang achterhaald is.
rationeel schreef op 5 februari 2013 12:40 :
[...]
Een tijdspanne van 10 jaar is veel te lang. De ontwikkelingen gaan zo snel, dat alles wat nu nieuw is dan al lang achterhaald is.
Als ze over 10 jaar nog zelfstandig aan de NPEX genoteert staat; maar wel 2,3 patenten/ goedkeuringen/ markt toetreding met producten verder zijn gekomen; vind ik dat je over je neus heen moet regeren. Het is niet zo irreëel dit te denken, en dan blijf ik liever participeren alsdat ik nu voor de midlange termijn >2 jaar een mooi rendement bereik. Dat is de strekking.
hierbij een bericht waarbij Uniqure milestones en royalties moet betalen over Glybera, ik overzie niet of dit betekent dat de intro dichtbij is, maar dat zal wel wishful thinking zijn.pharmalive.com/News/index.cfm?article...
Regulatory evaluation of Glybera in Europe — two committees, one mission Nature Reviews Drug Discovery (2013)doi:10.1038/nrd3835-c1 Published online 19 August 2013 Representing the first gene therapy to be approved in the Western world, alipogene tiparvovec (Glybera; Uniqure) has recently been said to have had a “substantial impact from a regulatory perspective” (Nature Rev. Drug Discov. 11, 664; 2012)1. The therapy was granted marketing authorization in the European Union for the treatment of lipoprotein lipase deficiency, which results in a clinically heterogeneous condition with a risk of potentially life-threatening pancreatitis2, at the end of 2012. The decision followed a positive opinion by the European Medicines Agency (EMA)'s Committee for Medicinal Products for Human Use (CHMP)3, and a previous recommendation of the EMA's Committee for Advanced Therapies (CAT)4, 5. The approval process for Glybera was extensively discussed in the scientific community, sometimes critically6, 7, 8, 9, 10, 11, 12. During the process, the opinions of the CHMP and the CAT differed: although the opinion of both committees was originally negative, in a “re-examination procedure” the opinion of the CAT became positive5, whereas the CHMP maintained its negative opinion13. However, both committees finally recommended approval of the medicine. As regulators who have been involved in this approval process, we would like to provide insight into why the Glybera procedure was challenging, and give assurance to the scientific community regarding confidence in both orphan drug and gene therapy regulation in Europe. In Europe, gene therapies undergo a centralized approval procedure via the EMA. For advanced therapy medicinal products (ATMPs), which include gene therapies, the CAT as an expert committee first performs a scientific assessment of the application dossier and prepares a draft opinion on its approvability for a final decision by the CHMP, a committee with considerable long-term expertise, which also ensures consistency in the opinions. The assessment process for Glybera was long and complex owing to multiple reasons. These included the complexity of the product class for which there was little previous regulatory experience (this was the first procedure for a gene therapy to correct a genetic deficiency); the long product development time, during which science evolved and specific regulatory requirements were about to be established; the complex disease scenario (a very rare disease) with a fluctuating clinical outcome (pancreatitis); and the fact that the company was small with academic origins (as usually also seen for other ATMPs)14.
The applicant's total clinical programme included 27 patients with lipoprotein lipase deficiency on a low-fat diet2. At first, the main measure of efficacy was based on a reduction in blood triglyceride levels. However, this was later changed to postprandial chylomicrons, as this biomarker was thought to more specifically address the pharmacodynamic effect of Glybera, whereas the effect on triglyceride levels was only short-lived. This raised additional issues during the scientific review: were the data robust enough for a previously non-validated biomarker (postprandial chylomicrons) in the presence of more inconclusive clinical evidence (pancreatitis)? Should data from that biomarker be accepted as pivotal evidence for activity, as it was scientifically better fitted to measure the treatment effect (as recommended by an ad hoc scientific advisory group to the CAT and CHMP) than the one originally defined in the protocol (triglyceride levels)? Or should one rather focus on that (formally failed) primary end point? There were only a handful of patients from the study population for whom such data on the newly proposed biomarker were available, and even fewer for a sustained period. Although different opinions were issued by the two committees during the procedure, both committees were consistent in their scientific judgement when the details were considered. The CHMP acknowledged the promising nature of the data by an almost unprecedented “positive tone” when drafting its initial negative opinion, so as to demonstrate that it would be prepared to reconsider the case once more specific and supportive data had been collected (and here the vast majority of the CHMP agreed). The CHMP was clearly of the view that development should continue. We consider that both committees had already, at that time, taken major steps towards finding a way for ATMPs to be used for the treatment of very rare diseases, by considering all data rather than single outcome measures. Both committees considered that evolution of scientific knowledge can make the appreciation of an emerging, biologically more plausible biomarker necessary, if well justified and supported by data. Both committees were well aware that the acceptance of a limited data set was a double-edged sword, as it could be perceived as lowering the standards. However, this was not the case: both committees agreed that the limited clinical efficacy and safety data set needed to be supplemented by additional data. The opinion of the two committees differed on the stage at which such additional data should be submitted: before approval (as initially preferred by the CHMP) or after approval (as recommended by the CAT). It is important to note that the final positive outcome was, to a major extent, also driven by an appreciation of the specific clinical scenario of lipoprotein lipase deficiency. Assessing orphan drugs in 'ultra-rare' conditions (here defined as a prevalence of less than 1 in 100,000) was not new to the CHMP; several drugs are already licensed for conditions with such low prevalence (Fig. 1). However, all of these conditions follow either a continuously progressive course of deterioration or a very active disease course if left untreated (Table 1). The effect of a therapeutic intervention for such conditions is therefore easier to measure within a relatively short timeframe.
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