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  1. [verwijderd] 2 oktober 2015 22:27

    flosz schreef op 1 oktober 2015 14:39:

    For $QURE fans: I just looked up Glybera price in German real world setting, €53.78k/vial, adj to weight ~€1.34M.

    Glybera met luid getoeter aangehaald als dure therapie toen Hillary haar tweet deed mbt dure medicijnen...maar ten eerste telt Glybera nog niet eens mee in de USA en het zijn verzekeraars in -Duitsland- die bereid zijn om dit te vergoeden. US panic for nothing! Denk niet dat Hillary in Duitsland gaat protesteren tegen de hoge prijs van Glybera...haar eigen politieke agenda betreft vnl de USA en zoals het politici wel vaker brengen: de soep wordt zelden zo heet gegeten als dat ze opgediend wordt.

    Dat BMY de kaspositie reeds riant gevuld heeft (en goed mogelijk nog veel verder zal aanvullen), met de resultaten met San F. kansen biedt voor veel meer toepassingen, daar staan beleggers waarschijnlijk niet bij stil.

    Ik vrees toch sterk voor een overname op korte termijn...positieve data voor Hemofilie kunnen voor een stroomversnelling zorgen, want een overnemer ziet de koers liever niet al te ver oplopen en zal daar vóór willen toeslaan.
  2. flosz 6 oktober 2015 17:21
    European Medicines Agency (EMA)CAT $QURE Glybera Publ.Oct.2,2015 P.7/8.

    * 2.11.  Type II variations
    * 2.11.1.  Glybera – alipogene tiparvovec; Orphan; EMA/H/C/002145/II/34 ?UniQure Biopharma B.V.; Scope: submission of final study report CT-AMT—011-02 ?Rapporteur: C. Niederlaender; CHMP Coordinator: G. Markey ?Action: for adoption ?Document(s) tabled:?Revised Assessment Report?Request for Supplementary Information ?The CAT Rapporteur presented the revised assessment reports for Glybera variation II/34. CAT adopted the RSI, requesting changes to the SmPC and PL. ?CAT adopted the response timetable

    * 2.11.2.  Glybera – alipogene tiparvovec; Orphan; EMA/H/C/002145/II/37-G?UniQure Biopharma B.V.; Scope: PI update section 4.8 and 5.1 (five years FU of final CSR ?study 011.01) and FU of 011.3 ?Rapporteur: C. Niederlaender; CHMP Coordinators: G. Markey
    Action: for adoption
    Document(s) tabled: Revised AR?RSI
    The CAT Rapporteur presented the revised assessment reports for Glybera variation II/37-G. CAT adopted the RSI, requesting changes to the SmPC and PL.
    CAT adopted the response timetable.

    2.11.3. Glybera – alipogene tiparvovec; Orphan; EMA/H/C/002145/II/38?UniQure Biopharma B.V.; Scope: PI update section 5.1 (final CSR study 011.05) (FU of
    Rapporteur: C. Niederlaender; CHMP Coordinator: G. Markey
    Action: for adoption
    Document(s) tabled: Revised AR?RSI
    The CAT Rapporteur presented the revised assessment reports for Glybera variation II/38. CAT adopted the RSI, requesting changes to the SmPC and PL.
    CAT adopted the response timetable.

    2.12. Other post-authorisation activities
  3. flosz 7 oktober 2015 02:24
    First gene therapy prescribed: How can we ensure there are more in the future?
    Author: James Smith, 10/06/15

    With contributions from blogger David Brindley

    September 2015 has been an historic month for regenerative medicine. For the first time ever in the Western world, a prescription has been issued for a gene therapy, Glybera. At the ESGCT and FSGT Collaborative Congress, Helsinki, this news was welcomed and spurred discussion on broader challenges in the gene therapy field.

    In 2012, alipogene tiparvovec, trade name Glybera (UniQure, Netherlands) became the first gene therapy to receive marketing authorization in the West, for the treatment of liprotein lipase deficiency in adults – a rare genetic disorder in which a person lacks a protein needed to break down fat molecules – with severe pancreatitis. However, various setbacks have resulted in a highly unusual delay between approval and first use as a marketed product. Across the whole of Europe, just 150 to 200 patients are eligible for treatment with Glybera; this, and numerous difficulties in commercializing the medicine, have resulted in an astounding and record breaking price tag of ~€1.1 million to treat a single patient.
    As we have previously discussed, pricing of potentially curative medicines is challenging. Gene therapies epitomize these challenges: not only do they have high cost of goods, but they are often used for diseases with small patient populations, which, somewhat ironically, become smaller still as patients are cured successfully. As noted by Alec Orphanidis, UniQure, prices for gene therapies must be triangulated according to these factors, and others such as benefits to society or reduced burden in the clinic in the long-term. As noted in a recent Nature Biotechnology editorial, the payer’s perspective must be considered.

    Aside from appropriate pricing, several valuable “key points to success in gene therapy” were raised by Nigel Parker, Chairman and CEO of FKD Therapeutics Oy, and former CEO of Ark Therapeutics, who led the first MAA for a gene therapy, Cerepro, in 2004.
    First, the gene therapy industry is undoubtedly populated by expert scientists doing exceptional science. The issue is that those who have the role of funding gene therapy companies and research may not be expert scientists. Too little attention is given to the need for effective communication of science into language that enables investors to evaluate it as a business opportunity, not just as exceptional science. (We commend CCRM for encouraging its bloggers to do just this.) Indeed, too little attention is given in the first place to the issue of whether the science actually has a clear, commercial application. If not, however great the science behind the therapy, it is doomed to fail.
    Second, the importance of aligning the company’s program with the appropriate funding model is pertinent. Raising venture capital is a well-trodden and often successful route in biotech, but there are downsides and alternatives. Timelines for return on investment for VC cash may be longer than desirable, particularly in the nascent and uncertain field of gene therapy. From the outset, companies seeking VC investment should recognize that a profitable exit is on the investor’s mind. Establishing partnerships may lead to more closely aligned interests, which could lead to a better chance of success. Ultimately, such decisions must be made on a case-by-case basis, but their importance should not be overlooked.
    Great science is not enough to build a great company. It is clear that great people, who can bridge the gap between science and finance, and communicate and think in both languages, are of pivotal significance. The CEO, in particular, will be responsible for decisions that will ultimately ‘make or break’ the business.
    When discussing gene therapies, the question “Is it worth it?” is often muttered. At such a high cost, can these products really be justified? Is it worth investing in what has proven historically to be a risky area? If there is a clear patient need addressed with appropriate business models, effective management, and exceptional science demonstrating clear, material results, this question will go away.

    The regenerative medicine community should be celebrating the first prescription of Glybera for the iconic achievement that it represents. However, while significant, it must be recognized that Glybera is just the ‘tip of the iceberg.’ As an orphan medicine and a gene therapy paving a largely unmapped regulatory route, pricing for Glybera, and the numerous challenges encountered in its commercialization, do not necessarily reflect the situation for future gene therapies, such as cell-based immunotherapies, expected to reach the market soon.
    Developers should learn from the challenges encountered in the pioneering approval of Glybera, and take heed of the advice outlined herein, to ensure that a healthy gene therapy industry can thrive and ultimately deliver more transformative medicines to patients who so desperately need them.
  4. bilbo3 19 oktober 2015 01:34
    Om het bericht van flosz te benadrukken, de eerste verkoop van glybera afgelopen september!!!

    September 2015 has been an historic month for regenerative medicine. For the first time ever in the Western world, a prescription has been issued for a gene therapy, Glybera. At the ESGCT and FSGT Collaborative Congress, Helsinki, this news was welcomed and spurred discussion on broader challenges in the gene therapy field
  5. Prof. Dollar 19 oktober 2015 08:31

    bilbo3 schreef op 19 oktober 2015 01:34:

    Om het bericht van flosz te benadrukken, de eerste verkoop van glybera afgelopen september!!!

    September 2015 has been an historic month for regenerative medicine. For the first time ever in the Western world, a prescription has been issued for a gene therapy, Glybera. At the ESGCT and FSGT Collaborative Congress, Helsinki, this news was welcomed and spurred discussion on broader challenges in the gene therapy field

    Bijna. Het recept is voorgeschreven door een arts... uiteraard heeft de patiënt ook een keuze.

    Maar inderdaad, zoals ook uit de woorden van Aldag viel op te maken, het lijkt erop dat ook de toediening recentelijk heeft plaatsgevonden. Het wachten is op een persbericht van Chiesi. Misschien wel deze week.
  6. flosz 29 oktober 2015 00:46
    Gentherapie senkt Fettgehalt des Blutes
    Weniger Sahne im Blut
    28.10.2015 17:09 Uhr
    Die Berliner Charité behandelt die erste Patientin in Europa mit der umstrittenen Gentherapie "Glybera"

    Ein wenig Fett hat jeder im Blut. Doch was in den Adern der 43-jährigen Patientin von Elisabeth Steinhagen-Thiessen fließt, das sei „eher wie Sahne“. 3000 Milligramm Triglycerid-Fette pro Deziliter Blut – gesund sei ein Wert von 150 bis 175 Milligramm, sagt die Internistin und Leiterin des Interdisziplinären Stoffwechselzentrums der Berliner Charité. Ursache ist ein seltener Erbgutdefekt, der den Abbau der Fette stört. Sie lagern sich nicht nur in masernartigen Pusteln in der Haut ab, sondern lösen auch extrem schmerzhafte Entzündungen der Bauchspeicheldrüse aus. „Über 40 Mal musste unsere Patientin deshalb schon auf der Intensivstation behandelt werden“, sagt die Ärztin.

    Erster Einsatz von Glybera in Europa
    Fünf bis sechs Prozent der Patienten mit dieser Lipoproteinlipase-Defizienz (LPLD) sterben vorzeitig oder leiden an Folgeerkrankungen wie Diabetes oder Bauchspeicheldrüsenkrebs. Eine Chance auf Heilung gab es bislang nicht. Bei der aus Süddeutschland stammenden Patientin zeigten auch die teuren wöchentlichen Blutwäschen, die das Fett aus dem Blut holen sollen, schon lange keine ausreichende Wirkung mehr. Deshalb entschloss sich Steinhagen-Thiessen für eine Gentherapie – die erste, die von den europäischen Behörden je zugelassen wurde.
    Der Wirkstoff Glybera (Alipogentoparvovec) besteht aus drei Billionen Kopien eines Gens, das bei LPLD-Patienten defekt ist oder fehlt. Es enthält die Information für ein Enzym des Fettstoffwechsels, eine Lipase, ohne die sich zu viel Fett im Blut anreichert. Entschärfte Viren dienen als Gentaxis und schleusen die Erbgutmoleküle in die Muskelzellen des Oberschenkels. Sie landen in den Zellkernen, werden aber nicht ins menschliche Erbgut eingebaut. Trotzdem produzieren sie das Enzym Lipase, das den Patienten fehlt.

    "Nicht quantifizierbarer Zusatznutzen"
    So zumindest die Hoffnung. Denn ob die Therapie, entwickelt von der niederländischen Biotechfirma Uniqure, tatsächlich wirkt, lässt sich nicht so einfach feststellen. Bei einer so seltenen Erkrankung wie LPLD, von der es in Europa kaum mehr als 20 bis 30 Patienten gibt, wäre eine Studie statistisch nicht aussagekräftig genug, selbst wenn alle Erkrankten weltweit daran teilnehmen könnten.

    Ein Dilemma für die Zulassungsbehörden. Daher – und auch damit Pharmafirmen überhaupt das wirtschaftliche Risiko wagen, Therapien für seltene Erkrankungen zu entwickeln – müssen Medikamente wie Glyberagegenüber den Zulassungsbehörden ihre Wirksamkeit nicht so rigoros nachweisen wie Arzneien gegen häufige Krankheiten. Trotzdem überzeugten die vorgelegten Daten von Uniqure die Europäische Zulassungsbehörde Ema, den Einsatz von Glybera zu empfehlen. Ungeachtet des hohen Preises von etwa 900 000 Euro – pro Patient

    Diese Kosten werden seit Mai auch von den gesetzlichen Krankenkassen in Deutschland erstattet. Denn aufgrund des Sonderstatus von Medikamenten für seltene Erkrankungen musste der zuständige Gemeinsame Bundesausschuss (G-BA) trotz der mageren Wirksamkeitsnachweise (nach einigem Hin und Her) Glybera einen „Zusatznutzen“ attestieren – allerdings nicht ohne zu betonen, dass er „nicht quantifizierbar“ sei.
    Gen-Effekt hält fünf Jahre an
    Die 43-jährige Patientin von Elisabeth Steinhagen-Thiessen bekam Glybera bereits vor vier Wochen – in Form von 20 Spritzen in die (vorher betäubten) Oberschenkel. Nennenswerte Nebenwirkungen seien nicht aufgetreten, sagt die Ärztin. Aber der „Sahnegehalt“ des Blutes sei drastisch gesunken: von 3000 auf 900 Milligramm. Zwar sei das noch immer viel mehr als bei Gesunden. Die Patientin müsse weiter eine unangenehme fett- und kohlenhydratarme Diät einhalten – „wenn auch vielleicht nicht mehr so streng wie früher“. Am wichtigsten sei aber, dass sie keine Bauchspeicheldrüsenentzündungen mehr befürchten müsse.
    Für Steinhagen-Thiessen ist das ein großer Erfolg. Zumal die Wirkung der eingeschleusten Gene den Erfahrungen nach „auf jeden Fall fünf Jahre anhält“. Das sei auch den hohen Preis wert. Denn bisher koste allein die Blutwäsche, zweimal pro Woche, jedes Mal 1000 Euro. Von den Kosten für ständige, im Jahresrhythmus wiederkehrende intensivmedizinische Behandlungen erneuter Bauchspeicheldrüsenentzündungen ganz zu schweigen. „Wir sind damit in einem Bereich, wo sich der Einsatz nicht nur medizinisch, sondern auch volkswirtschaftlich rechnet“, sagt Ulrich Frei, Ärztlicher Direktor der Charité.
  7. DrMedicalValue 29 oktober 2015 11:03
    Nou als ik via het forum kon applaudiseren, dan was het voor Flosz! Great catch! Ook zo fijn om te lezen over die patient...

    @Prof: Nou moet Chiesi snel komen met PR. Nogmaals: zij doen in mijn ervaring 1x per jaar een company update met de jaarcijfers. Misschien nu een extra tussendoortje om $QURE een plezier te doen.

    Hopelijk gaan er nu meer landen vergoeden, maar ik zou zo snel niet weten welke. Italie is een kanshebber: zijn pro gentherapie. Hebben in CAT en CHMP Glybera altijd gesteund. En Chiesi hometurf. Makes a difference in Italy.

    Chiesi= 'private' Italiaanse club (daar kennen we er meer van!). Slimme familie. Hemo B rechten voor Europa voor een prikkie gekocht. Entrepreneurs & $QURE gered. Respect.

    Naar wie gaan de rechten voor de USA voor hemo B? Zou wel eens de next deal kunnen worden...
  8. Prof. Dollar 29 oktober 2015 11:53
    @DrMedicalValue: Ik heb de vraag uitgezet bij de communicatieadviseur van Chiesi. Ben benieuwd of die reageert. Anders is het, wat bilbo3 ook zegt, wachten tot kwartaal 3 publicatie medio eind november/begin december (bevat meestal ook company update).

    Over de rechten voor de USA voor hemo B... Chiesi heeft meerdere rollen: co-developer (funding), commercialisatie en distributie. Mogelijk is alleen die laatste van toepassing in de USA of het wordt een combi met hemo A. Laten we eerst de resultaten maar eens afwachten. ;-)
  9. bilbo3 29 oktober 2015 13:28
    Spritzen gegen Gendefekt

    Europaweit erste Gentherapie erfolgreich angewendet

    An der Charité – Universitätsmedizin Berlin ist jetzt europaweit die erste Patientin mit einer medikamentösen Gentherapie gegen eine seltene Stoffwechselerkrankung behandelt worden: die Erbkrankheit Lipoproteinlipase-Defizienz (LPLD). Die Erkrankung führt immer wieder zu schmerzhaften Bauchspeicheldrüsenentzündungen, die so gravierend sein können, dass die Patienten intensivmedizinisch versorgt werden müssen.

    Den Betroffenen fehlt aufgrund eines Gendefekts das Enzym Lipoproteinlipase (LPL), das für den Abbau von Blutfetten zuständig ist. Bisher galt die Stoffwechselerkrankung LPLD als unheilbar, teilweise führte sie zu einem frühen Tod. Das neuartige Medikament Glybera ermöglicht nun eine Gentherapie: Gesunde Kopien des LPL-Gens werden dem Patienten unter Narkose in die Oberschenkelmuskulatur injiziert. Der Körper wird damit in die Lage versetzt, das bisher fehlende Enzym selbst zu produzieren. Der Gendefekt kann also mit einer einmaligen Therapie behoben werden.

    Die Patientin hat die Therapie an der Charité gut vertragen: Nach nur sechs Wochen waren ihre Blutfettwerte deutlich gesunken – ein Hinweis darauf, dass ihr Körper das Enzym LPL nun selbst produzieren kann. Langfristig geht es für die Betroffenen um die Verminderung von Bauchspeicheldrüsenentzündungen. In klinischen Studien konnte hier ein Rückgang um 40 bis 50 Prozent gezeigt werden.

    Prof. Dr. Elisabeth Steinhagen-Thiessen, Leiterin des Interdisziplinären Stoffwechsel-Centrums, behandelte die erste Patientin mit der innovativen Methode: „Für uns stellt diese Behandlungsform den Durchbruch in ein neues therapeutisches Zeitalter dar: die einmalige, kausale Therapie von Gendefekten.“ Sie ergänzt: „Die Behandlung darf nur in Spezialzentren durchgeführt werden, die eine Expertise im Umgang mit Zelltherapeutika vorweisen können. In Deutschland hat sich das interdisziplinäre Lipid- und Stoffwechselzentrum der Charité dafür qualifiziert.“
  10. flosz 11 november 2015 10:43
    Finally, I’ll provide a very quick update on Glybera which is the first product whose active ingredients was derived from our platform to have received commercial approval. It’s currently the only gene therapy product to receive approval in Europe. Xenon Pharmaceuticals' (XENE) CEO Simon Pimstone on Q3 2015 Results - Earnings Call Transcript
    Nov. 10, 2015 10:46 PM ET  

    Glybera is specifically indicated for the treatment of a subsets of adult patients diagnosed with an orphan lipid disorder known as Lipoprotein lipase deficiency or LPLD, which is confirmed by genetic testing. These patients suffer from severe multiple pancreatitis events and this is despite dietary fat restrictions.
    Glybera was developed by our licensee uniQure Biopharma, and it’s being commercialized by uniQure’s partner Chiesi. Although we are not giving specific guidance about Glybera, we understand that Chiesi has submitted price and reimbursement dossiers in key European countries to make Glyberia accessible to patients. We will certainly provide additional information as we receive it.

    And lastly, we anticipated a commercial launch of Glybera in the EU by our licensee uniQure’s commercial partner Chiesi
  11. flosz 28 november 2015 12:14
    Bringing regenerative medicines to the clinic: the future for regulation and reimbursement

    Current practices and reform proposals for the regulation of advanced medicinal products in Canada

    Regulators, Researchers Seek to Advance Research on Cellular & Gene Therapies

    Glybera EMA(publ.27-11-'15)Procedural steps taken&scientific info after authorisation
  12. flosz 5 april 2016 00:15

    Our clinical development program for Glybera to date has consisted of three non-controlled, prospective, open-label clinical trials in which we administered Glybera to a total of 27 LPLD patients. In addition, we carried out two retrospective case note reviews of 19 of the 27 patients to determine the impact of Glybera treatment on the frequency and severity of pancreatitis events up to 6 years post-gene therapy. we observed Although consistent reduction in total tryglyceride levels were not obtained in the 3 prospetive trials, a a significant improvement in the appearance and removal of newly formed chylomicrons was observed at week 52 after Glybera, the last time point tested in the last prospective study. The retrospective chart review in patients who participated in the clinical trials also provided evidence of clinical benefit in the form of a reduction of approximately 40-60% in pancreatitis events and in the severity of attacks, as measured by ICU admissions.
    To fulfill the key conditions of the approval of Glybera by the EMA we are required to implement a patient registry prior to commercial launch and to conduct post-approval clinical trials of Glybera.
    The patient registry was put in place in May 2014. In 2015 we completed a non-interventional healthy volunteer study to establish post-prandial chylomicron clearance test curves in 8 normal individuals following fat-containing standardized meal. We currently plan to enroll 12 patients with LPLD into a phase IV study. We anticipate that the trial will be conducted as a multicenter trial including sites in the United States and Canada. The EMA has approved an initial protocol for this clinical trial in 12 patients.
    We also developed an improved manufacturing process for Glybera, which addresses also our post-approval commitments and received EMA approval in January 2016.

    Glybera Commercialization
    To obtain payment coverage for Glybera from the relevant pricing and reimbursement agencies in countries in the European Union, Chiesi must generally submit price and reimbursement dossiers to the relevant bodies in each country. In Germany, Glybera has received its first published price in November 2014. The pricing model chosen for Glybera in Germany is a one-time payment of EUR 41,000 per vial. A single treatment for patients weighing 60-70 kilograms requires 20-24 vials.
    Following an assessment by the German Federal Joint Committee (Gemeinsamer Bundesausschuss, or G-BA), the G-BA has agreed with Chiesi in October 2015 that Glybera, given the very small patient population and highly specialized administration process and requirements, should no longer be considered for national reimbursement, but rather will be handled as a hospital-only product. This means that reimbursement for all future patients should be obtained via a single-case reimbursement request made by the treating physician to the appropriate sick-fund (as was done for the first patient treated in September 2015).

    In France, the Haute Autorité de Sante (HAS) informed Chiesi in mid-2015 that Glybera was not considered by them to provide sufficient benefit to patients to be considered for reimbursement by the national health program. Following an appeal by Chiesi in November 2015, the HAS maintained their initial conclusion.

    On April 1, 2015, a list price was established in the United Kingdom at a similar level to the list price in Germany. Pricing and reimbursement decisions are made on a country-by-country basis in the European Union and no country is under the obligation to follow another's pricing; however, prices in one country can influence the price level in other countries. We expect that reference prices in the larger countries in the European Union will provide a basis for pricing discussions in other countries in the European Union.
  13. flosz 4 mei 2016 08:23
    The World’s Most Expensive Medicine Is a Bust
    The first gene therapy approved in the Western world costs $1 million and has been used just once. The doctor who tried it says the price is “absolutely too high.”
    by Antonio Regalado May 4, 2016
    The most expensive drug in history is a money loser that’s not reaching patients. In fact, it’s only been paid for and used commercially once since being approved in 2012.

    The medication in question is alipogene tiparvovec, better known as Glybera, a medicine widely heralded as the “first gene therapy” in the Western world and whose approval helped ignite an explosion of investment and excitement around treatments that correct DNA.

    But when the Berlin physician Elisabeth Steinhagen-Thiessen wanted to give a patient Glybera last fall, it wasn't so easy. She says she had to prepare a submission as thick as “a thesis” for German regulators and then personally call the CEO of DAK, one of Germany’s large sickness funds, or insurers, to ask him to pay the $1 million price tag.

    Last September, she gave 40 injections to the muscles of a 43-year-old woman with an ultra-rare disease called lipoprotein lipase deficiency. Such patients don’t process fat correctly. “You draw blood and you are astonished, there is no red blood, it's cream,” Steinhagen-Thiessen says. One symptom is debilitating abdominal pain. Her patient had been hospitalized more than 40 times.

    A dose of Glybera contains trillions of viruses harboring correct copies of the lipoprotein lipase gene. And Steinhagen-Thiessen says the treatment, at Charite Hospital in Berlin, was a success. The woman hasn’t been back to the emergency room since the treatment and is now “living like you and me.”

    But this single use of the drug just proves that Glybera is a flop. The problem is its staggering million-dollar price tag, too few patients, and questions about how effective it is.

    The company that developed Glybera, UniQure, based in Amsterdam and Lexington, Mass., last fall dropped plans to get it approved in the U.S. and has turned over European sales to the Italian drug maker Chiesi Farmaceutici, which calls selling the drug "challenging."

    “I think we learned a tremendous amount about what to do and what not to do, but commercially it has not been a success. It still drains a lot from the company,” says Dan Soland, UniQure's CEO. UniQure is now focused on developing other gene therapies, including one to treat hemophilia.

    How Glybera turned into a money loser is a cautionary tale for gene therapy, a resurgent technology that has been drawing investor interest because of its promise to cure rare, inherited diseases with one-time repairs to a person’s DNA. A single dose of gene therapy can change the genetic instructions inside a person's cells in ways that last many years, or even a lifetime.

    In addition to Glybera, there is at least one form of gene therapy approved in China to treat cancer by adding a gene to tumors, and late last year Amgen won U.S. approval for Imlygic, which uses the herpes virus to shrink skin cancers.

    But reversing inherited genetic disease remains gene therapy's great promise. And more treatments will reach the market soon. In April, European authorities gave a preliminary green light for a gene therapy for severe combined immune deficiency, to be sold by GlaxoSmithKline. And by 2017, handicappers expect, a Philadelphia company called Spark Therapeutics could win approval in the U.S. for a gene fix that partly reverses one form of blindness. Like the metabolic condition Glybera treats, both these diseases are incredibly rare. Glaxo estimates that only 14 cases of severe combined immune deficiency come to light each year in Europe.

    The combination of rare diseases with a costly new technology that needs to be used only once is what could lead to exorbitant prices. Analysts have said Spark’s treatment might cost $500,000 per eye. And with nearly 670 gene-therapy trials under way, and 68 in the later stages, known as Phase III, it’s becoming “urgent” to understand how these therapies will be paid for, says Morrie Ruffin, a consultant with the Alliance for Regenerative Medicine, a trade group. The current system “was not established with these types of products in mind,” he says.

    Executives at Glaxo say they don’t expect to charge anything near a million dollars. But with $35 billion a year in revenue, they don’t need to rely on gene therapy for profits. “From a pure business, money-making perspective, it’s a challenge,” says Sven Kili, head of gene-therapy development at Glaxo. “It’s a single treatment that lasts indefinitely and patients don’t come back. And there aren’t many of them. It is not something that would make a venture capitalist jump with joy.” He says Glaxo is thinking of a price “way below” what people might expect for once-in-a-lifetime therapy and “nowhere near” that of Glybera.
  14. flosz 4 mei 2016 08:24
    There isn’t much doubt that Glybera’s approval in 2012 was important. It showed that gene therapy, once branded as too risky, could be safe and was ready to be commercialized. Since then, a large number of new companies have been formed and more pharmaceutical firms have shown interest. Piper Jaffray, the investment bank, says 2015 was the most “momentous” year yet for gene therapy. About $2 billion was raised by 10 public gene-therapy companies in the U.S. last year.

    “No one knew whether this was going to be a viable strategy,” says David Schaffer, an expert on gene-therapy viruses at the University of California, Berkeley, who is also on UniQure’s board. “It was just a huge boost for the field to know someone got something over the finish line. “

    UniQure started out as Amsterdam Molecular Therapeutics, a 1998 spin-off from the University of Amsterdam. Doctors there had zeroed in on an enzyme, lipoprotein lipase, that muscles produce and which digests fat in the blood. In some people, both copies of the gene to make the enzyme are mutated, and don’t work correctly.

    The Amsterdam company spent more than $100 million testing the drug and carving a path through Europe’s medical rules and regulations, which weren’t geared to consider a new technology like gene therapy. Initially, for instance, regulators said they expected a clinical trial of 342 patients. Executives wryly noted that there were only 200 people with the disease in all of Europe. “The filing was a nightmare experience,” says Sander van Deventer, a biotech investor who was once the company’s chief medical officer. “They didn’t have the know-how to approve an advanced therapy.”

    But the data for Glybera wasn’t rock-solid either. The drug was given to just 27 people in three “open-label” experimental studies, meaning no patient got a placebo. Those tests never showed a lasting change in fat levels in their blood—though the company argued that people who got the drug had fewer episodes of pancreatitis, the painful complication of the disease.

    By 2012, the company had failed twice to convince European regulators. It decided to reorganize, incorporating as UniQure, and mounted a last, successful attempt to get the drug approved. “We didn’t want to give it up even though the commercial outlook was not good,” says Van Deventer.

    A year after the approval by Europe’s top medicines' body, UniQure was able to go public on the Nasdaq, raising $82 million. Although it called the commercialization of Glybera a top priority, by last fall UniQure instead chose to scrap its plans to sell the drug in the U.S. after the Food and Drug Administration said new, expensive trials would be needed.

    Nor has Glybera convinced the national regulators in Europe who decide what drugs get reimbursed. Last year, French authorities said they would not pay for the drug. Germany judged Glybera’s benefits “non-quantifiable.” It leaves doctors and insurers to make decisions on a case-by-case basis. In the Netherlands, where the technology was invented and supported by R&D grants, the health minister complained that Glybera was part of a pattern of price gouging by drug companies.

    The decision to charge around $1 million for Glybera (the exact amount depends on the patient’s weight) does raise questions. Some people think gene therapies should be paid for in yearly installments, and only so long as they keep working. But that wasn’t possible with Glybera because there was no clear-cut way to track the effects of the drug. “There is pressure to pay all at once—the payers said they wanted to pay up front,” says Van Deventer. “But that created a lot of anger in Europe. It’s a shame. It became the 'one-million' therapy. There is an anti-innovation climate and people don’t want to pay for it at all.”

    Even modest expectations for Glybera’s sales now look much too rosy. (Early last year, one analyst projected peak revenues of $57 million a year.) Part of the reason is that the disease is so rare, just one in a million people, that it’s often not diagnosed correctly, and finding customers is hard work. Following its reorganization, UniQure sold European marketing rights to Chiesi, a small Italian drugmaker with little experience selling such a complex therapy.

    That means even the few patients who want the West’s first gene therapy can’t easily get it. Steinhagen-Thiessen says she knows of a man in Luxembourg and a family in Czechoslovakia who are interested, but they don’t yet have a way to pay for it. A Chiesi spokesperson says it is working to create a registry of patients in Europe.

    Eventually, the German insurer DAK did pay did pay 900,000 euros, or about $1 million, to cover the cost of treating Steinhagen-Thiessen’s patient. Most of that went to pay for Glybera. “I think the price is absolutely too high. I am not sure that other insurance companies are willing to pay that,” says the doctor. “From an ethical point of view we should have a lower price. I don’t think the companies can make a profit by it in any case.”

    Schaffer, the Berkeley professor, says gene-therapy companies still have work to do demonstrating their concepts. It may be too soon to turn a profit. “I think that the field should focus on disease targets which are well known, where there is a big chance for medical success, whether or not there is commercial success,” he says. “People who are investing in early stage biotechs are doing it because of the promise of the future, not immediate returns tomorrow.”
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