25 août 2009
EMEA 14-08-09 : PQuestions et réponses sur les manques de Cerezyme et Fabrazyme (virus Vesivirus 2117)
Vous trouverez des articles traitant du même sujet dans la catégorie "EMA".
Liens utiles à la fin des catégories.
Ghislaine SURREL
maladies-lysosomales-subscribe@yahoogroupes.fr
European Medicines Agency
7 Westferry Circus, Canary Wharf, London E14 4HB, UK
Tel. (44-20) 74 18 84 00 Fax (44-20) 74 18 84 16
E-mail: mail@emea.europa.eu http://www.emea.europa.eu
European Medicines Agency, 2009. Reproduction is authorised provided the source is acknowledged.
London, 14 August 2009
Doc. Ref. EMEA/510766/2009
Questions and answers on the shortages of Cerezyme and Fabrazyme
The European Medicines Agency has reviewed treatment recommendations from June 2009 on which
patients should receive Cerezyme and Fabrazyme as a priority during the shortage of these two
medicines over the next few months. The shortage, which is expected to last until the end of the year,
is happening because of a problem in a factory where the active substances for the medicines are
made.
The company has informed the Agency that the supply shortage of Cerezyme is more severe than it
previously thought. Because of this, the Agency is now recommending the following:
• for Cerezyme, only patients at greatest need of treatment will receive Cerezyme but at a reduced
dosage;
• for Fabrazyme, the recommendations given in June 2009 remain the same. They are ensuring that
patients at greatest need of treatment continue to receive this medicine until the shortage is
resolved.
What are Cerezyme and Fabrazyme?
Cerezyme and Fabrazyme are medicines that are used in two rare, inherited, life-threatening diseases
in which patients have a lack of an enzyme involved in the breakdown of fatty substances in the body:
• Cerezyme is used in patients with Gaucher disease, a disease in which patients do not have enough
of an enzyme called alglucerase. Cerezyme contains imiglucerase, which is a copy of the natural
enzyme;
• Fabrazyme is used in patients with Fabry disease, a disease in which patients do not have enough of
an enzyme called alpha-galactosidase A. Fabrazyme contains agalsidase beta, which is a copy of
the natural enzyme.
In both medicines, the replacement enzyme is made by a method known as ‘recombinant DNA
technology’: the enzymes are made by cells that have received genes (DNA) that makes them able to
produce the enzymes. The cells are grown in special tanks called ‘bioreactors’ over a three- to
four-month process, and the enzyme is extracted from the culture at regular intervals during the
process.
Cerezyme has been authorised since November 1997 and Fabrazyme since August 2001. Both
medicines are marketed in all Member States of the European Union.
What is the problem with Cerezyme and Fabrazyme?
Earlier this year, Genzyme, the company that makes Cerezyme and Fabrazyme, became aware of
reduced yields from the bioreactors used to produce Cerezyme and Fabrazyme at their production site
in Allston Landing in the United States of America. The company found out that the bioreactors were
contaminated with a virus (a calicivirus of the type Vesivirus 2117). This virus is not known to cause
disease in humans, but it can attack the cells used to produce these medicines. The contamination has
an impact on cell growth, affecting the quantity, but not the quality, of the enzymes produced by the
cells.
In June 2009, in order to sanitise the production facilities and conduct an investigation to prevent the
contamination from re-occurring, the company had to stop the production of new batches of Cerezyme
and Fabrazyme for an extended period of time. As a result of the supply shortage of both medicines,
the company, in agreement with the Agency, recommended some temporary changes to the way
Cerezyme and Fabrazyme were prescribed and used.
Although manufacturing is resuming, Genzyme has now informed the European Medicines Agency
that the stocks of Cerezyme are lower than it said they were in June. Therefore the recommendations
on the use of Cerezyme have had to be revised. These changes should be implemented immediately.
What are the recommendations while the shortages are ongoing?
Revised recommendations for Cerezyme
For Cerezyme, priority is given to infants, children and adolescents, and adults with severe, life-
threatening disease progression:
• infants, children and adolescents should receive Cerezyme at a reduced dose or at a reduced
infusion frequency. However, no patient should be treated at a dose lower than 15 units per
kilogram body weight every two weeks, or alternative treatment should be considered.
• adult patients with severe, life-threatening disease progression should receive Cerezyme at a
reduced dose or at a reduced infusion frequency. However, no patient should be treated at a dose
lower than 15 U/kg every four weeks, or alternative treatment should be considered.
In adult patients without severe, life-threatening disease progression, alternative treatment such as
miglustat should be considered or treatment should be interrupted. Adults who demonstrate
progression to severe, life-threatening disease should re-initiate treatment with Cerezyme.
Recommendations for Fabrazyme
For Fabrazyme, as in June 2009, priority is given to children and adolescents, and adult male
patients, who should continue to receive Fabrazyme as one infusion every two weeks.
However, adult female patients, in whom the disease is less severe, may receive Fabrazyme at a
reduced dose.
All patients will be closely monitored while treatment is suspended or while they are receiving
reduced doses of Cerezyme or Fabrazyme. Reporting of side effects will continue as normal, with
doctors recording the batch numbers of the medicines in each patient’s records.
These changes will need to continue until end of 2009 when the shortages are to be resolved.
What are the recommendations for prescribers?
• Doctors who look after patients with Gaucher or Fabry disease should be aware of the shortages,
and should consider which patients should be switched to the reduced dose or other treatment.
What are the recommendations for patients with Gaucher disease who receive Cerezyme?
• Young patients with the disease (infants, children and adolescents) and adult patients with severe,
life-threatening disease progression should be contacted by their doctor to discuss their treatment
options. While the shortages are ongoing, they may be treated at a different frequency and with a
reduced dose.
• Adult patients without severe, life-threatening disease progression should be contacted by their
doctor to discuss the possibility of switching to alternative treatments (e.g. miglustat) or suspending
treatment.
• Patients who have any questions should speak to their doctor or pharmacist.
What are the recommendations for patients with Fabry disease who receive Fabrazyme?
• There are no consequences for young patients with the disease (infants, children and adolescents)
or for adult male patients.
• Adult female patients with Fabry disease should be contacted by their doctor to discuss their
treatment options. While the shortages are ongoing, they may be treated at the same frequency
(every two weeks) but with a reduced dose.
• Patients who have any questions should speak to their doctor or pharmacist.
What will happen next?
Genzyme is sending specific communications to all Cerezyme prescribers on how to select patients for
dose reduction, switch to alternative treatment or suspension of treatment, according to the new
recommendations. Genzyme has informed the Agency that no changes to the June recommendations
are required for Fabrazyme.
Genzyme has also informed the Agency that these stock-sparing measures will have no impact on the
supplies to ongoing clinical trials.
The European Medicines Agency will update this document as new information becomes available.
07 juillet 2009
EMEA 25/06/09 : Questions et réponses sur la gestion des stocks de Cerezyme et Fabrazyme (vesivirus 2117)
Vous trouverez des articles traitant du même sujet dans la catégorie "EMA".
Liens utiles à la fin des catégories.
Ghislaine SURREL
maladies-lysosomales-subscribe@yahoogroupes.fr
European Medicines Agency EMEA
London, 25 June 2009
Doc. Ref. EMEA/388773/2009
Questions and answers on the shortages of Cerezyme and Fabrazyme
The European Medicines Agency has recommended which patients should receive Cerezyme and Fabrazyme as a priority during the expected shortage of these two medicines over the next few months. The shortage is happening because of a problem in the factory where the active substances for the medicines are made. The recommendation is intended to ensure that the patients at greatest need of treatment continue to receive these medicines while the company solves its supply problems.
What are Cerezyme and Fabrazyme?
Cerezyme and Fabrazyme are medicines that are used in two rare, inherited, life-threatening diseases in which patients have a lack of an enzyme involved in the breakdown of fatty substances in the body:
• Cerezyme is used in patients with Gaucher disease, a disease in which patients do not have enough of an enzyme called alglucerase. Cerezyme contains imiglucerase, which is a copy of the natural enzyme;
• Fabrazyme is used in patients with Fabry disease, a disease in which patients do not have enough of an enzyme called alpha-galactosidase A. Fabrazyme contains agalsidase beta, which is a copy of the natural enzyme.
In both medicines, the replacement enzyme is made by a method known as 'recombinant DNA technology': the enzymes are made by cells that have received genes (DNA) that makes them able to produce the enzymes. The cells are grown in special tanks called 'bioreactors' over a three- to four-month process, and the enzyme is extracted from the culture at regular intervals during the process.
Cerezyme has been authorised since November 1997 and Fabrazyme since August 2001. Both medicines are marketed in all Member States of the European Union.
What is the problem with Cerezyme and Fabrazyme?
Earlier this year, Genzyme, the company that makes Cerezyme and Fabrazyme, became aware of reduced yields from the bioreactors used to produce Cerezyme and Fabrazyme at their production site in Allston Landing in the United States of America. The company have found that the bioreactors were contaminated with a virus (a calicivirus of the type Vesivirus 2117). This virus is not known to cause disease in humans, but it can attack the cells used to produce these medicines.
The contamination has an impact on cell growth, affecting the quantity, but not the quality, of the enzymes produced by the cells. All batches prepared using enzymes produced prior to the contamination being detected have been tested and the Agency has confirmed that they are suitable for release onto the market.
The company has now shut down the factory so that it can be sanitised. While this is ongoing, Genzyme cannot make any new batches of Cerezyme or Fabrazyme. Existing stocks are expected to run out as early as August 2009 for Cerezyme, and by October 2009 for Fabrazyme. To ensure that these stocks last as long as possible while the company resolves its manufacturing problems, the company, in agreement with the European Medicines Agency, is recommending some temporary changes to the way Cerezyme and Fabrazyme are prescribed and used. These changes should be implemented immediately.
What are the recommendations while the shortages are ongoing?
• Cerezyme For Cerezyme, priority is given to infants, children and adolescents, and adults with active disease progression. These patients can continue to receive Cerezyme at the standard dose schedule of one infusion every two weeks. However, adult patients without clinical evidence of active disease progression should receive Cerezyme at a reduced dose (half a dose once every two weeks) or at a reduced infusion frequency (once a month at their current dose).
• Fabrazyme For Fabrazyme, priority is given to children and adolescents, and adult male patients, who should continue to receive Fabrazyme as one infusion every two weeks. However, adult female patients, in whom the disease is less severe, may receive Fabrazyme at a reduced dose. All patients will be closely monitored while they are receiving reduced doses of Cerezyme or Fabrazyme. Reporting of side effects will continue as normal, with doctors recording the batch numbers of the medicines in each patient's records.
These changes will need to continue until the shortages are resolved, by the end of the year at the latest.
What are the recommendations for prescribers?
• Doctors who look after patients with Gaucher or Fabry disease should be aware of the shortages, and should consider which patients should be switched to the reduced dose.
What are the recommendations for patients with Gaucher disease?
• There are no consequences for young patients with the disease (infants, children and adolescents) or for adult patients with active disease.
• Adult patients with Gaucher disease whose disease is not currently active should be contacted by their doctor to discuss their treatment options. While the shortages are ongoing, they may be treated at the same frequency (every two weeks) but with a reduced dose, or they may be asked to come to the clinic only once a month. • Patients who have any questions should speak to their doctor or pharmacist.
What are the recommendations for patients with Fabry disease?
• There are no consequences for young patients with the disease (infants, children and adolescents) or for adult male patients.
• Adult female patients with Fabry disease should be contacted by their doctor to discuss their treatment options. While the shortages are ongoing, they may be treated at the same frequency (every two weeks) but with a reduced dose. • Patients who have any questions should speak to their doctor or pharmacist.
What will happen next?
Genzyme is sending specific communications to all Cerezyme and Fabrazyme prescribers to ensure that they have full details on how to select patients for dose reduction. Genzyme has also informed the CHMP that these stock-sparing measures will have no impact on the supplies to ongoing clinical trials. While the company is sanitising its factory, it is also investigating in depth, at the request of the Agency, the reasons why the contamination arose. The company will keep the Agency informed of its findings.
The European Medicines Agency will update this document as new information becomes available.
26 juin 2009
EMEA 25-06-09 : Problèmes d'approvisionnement de Cerezyme et Fabrazyme suite contamination d'1 réacteur (virus Vesivirus 2117)
Vous trouverez des articles traitant du même sujet dans la catégorie "EMA". Liens utiles à la fin des catégories. Ghislaine SURREL maladies-lysosomales-subscribe@yahoogroupes.fr
European Medicines Agency Press office
London, 25 June 2009
Doc. Ref. EMEA/389995/2009
PRESS RELEASE
Supply shortages of Cerezyme and Fabrazyme - priority access for patients
most in need of treatment recommended
The European Medicines Agency's (EMEA) Committee for Medicinal Products for Human Use
(CHMP) has recommended that patients who are in greatest need of treatment are given priority access to Cerezyme (imiglucerase) and Fabrazyme (agalsidase beta) during the expected supply shortage of these two medicines over the next few months. Cerezyme and Fabrazyme are both used to treat rare, inherited enzyme-deficiency disorders. Cerezyme is used in patients with Gaucher disease, a disease in which patients do not have enough of an enzyme called alglucerase. Fabrazyme is used in patients with Fabry disease, a disease in which patients do not have enough of an enzyme called alpha-galactosidase A.
The supply shortage is caused by the shutting down of Genzyme's production site in Allston Landing, in the United States of America, where both medicines are produced. The company found a viral contamination (calicivirus of the type Vesivirus 2117) and has shut down the production facility for a sanitization of the bioreactors. The virus is not known to cause disease in humans, but it may affect the quantity, but not the quality, of the enzymes produced in the bioreactors. An in-depth investigation of the cause of the contamination is ongoing. While the facility is shut down, no new stocks of Cerezyme and Fabrazyme can be produced. All batches manufactured prior to the detection of the contamination were tested and the Agency confirmed that they are suitable for release. To ensure that existing stocks last as long as possible until new batches can be produced, the European Medicines Agency has agreed to some temporary changes to the way these medicines are prescribed proposed by the company. These changes should be implemented immediately.
For Cerezyme, priority is given to infants, children and adolescents, and adults with active disease progression. These patients can continue to receive Cerezyme at the standard dosage schedule of one infusion every two weeks. However, adult patients without clinical evidence of active disease progression should receive Cerezyme at a reduced dose (half a dose once every two weeks) or at a reduced infusion frequency (once a month at their current dose).
For Fabrazyme, priority is given to children and adolescents, and adult male patients, who should continue to receive Fabrazyme as one infusion every two weeks. However, adult female patients, in whom the disease is less severe, may receive Fabrazyme at a reduced dose.
These are temporary recommendations and do not change the currently approved Product Information for either Cerezyme or Fabrazyme. It is expected that these changes will need to continue until the end of the year.
Notes:
1. More information is available in a question-and-answer document.
2. More information on Cerezyme, including the currently approved Product Information, is available in the European Public Assessment Report:
http://www.emea.europa.eu/humandocs/Humans/EPAR/cerezyme/cerezyme.htm.
More information on Fabrazyme, including the currently approved Product Information, is available in the European Public Assessment Report:
http://www.emea.europa.eu/humandocs/Humans/EPAR/fabrazyme/fabrazyme.htm
3. This press release, together with other information on the work of the EMEA, can be found on the EMEA website: www.emea.europa.eu Media enquiries only to: Martin Harvey Allchurch or Monika Benstetter Tel. (44-20) 74 18 84 27, E-mail press@emea.europa.eu
7 Westferry Circus, Canary Wharf, London, E14 4HB, UK
Tel. (44-20) 74 18 84 00 Fax (44-20) 74 18 84 09
E-mail: mail@emea.europa.eu
http://www.emea.europa.eu
29 août 2005
La Commission européenne envisage des prix conseillés pour les médicaments orphelins
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Article classé dans la catégorie "EMA".
Enfin un espoir pour les patients de Gaucher des pays de l'Est qui ne peuvent pas être traités. Liens utiles à la fin des catégories
|
Le 22/08/2005 |
APM |
LONDRES, 19 août (APM Santé) - La Commission européenne est en train d'examiner des propositions visant à établir des prix conseillés pour les médicaments orphelins, afin de réduire les délais d'accès à ces produits, a indiqué à APM Santé l'organisation européenne Eurordis. |
LONDRES, 19 août (APM Santé) - La Commission européenne est en train d'examiner des propositions visant à établir des prix conseillés pour les médicaments orphelins, afin de réduire les délais d'accès à ces produits, a indiqué à APM Santé l'organisation européenne Eurordis. Eurordis, qui regroupe des associations de patients concernés par des maladies rares, a indiqué qu'elle collaborait avec la Commission européenne pour développer cette initiative, qui prévoit la création d'un comité de "transparence sur les médicaments orphelins" qui fixera des prix pour les nouveaux médicaments orphelins. Ces prix seront fixés en fonction des données d'efficacité et de sûreté. La valeur thérapeutique ajoutée démontrable, sur laquelle se seront mis d'accord le comité et les fabricants - tels que la réduction des entrées à l'hôpital - pourrait être prise en compte dans ce prix de départ, qui fera ensuite l'objet de négociations finales entre le fabricant et chaque Etat, selon Eurordis. François Houyez, l'un des responsables d'Eurordis, a précisé dans un entretien avec APM Santé que cette initiative permettrait de réduire la période de négociation sur les prix des médicaments orphelins dans l'Union européenne. "Certains pays attendent les décisions de cinq autres pays sur les prix, et ils utilisent une moyenne de ces prix comme base de leurs négociations. Imaginez les délais que cela peut produire", a-t-il expliqué. François Houyez a indiqué que soit les patients ayant besoin de ces produits orphelins doivent attendre, soit les fabricants fournissent le traitement avant qu'un prix soit fixé et doivent ensuite faire face à un délai pouvant aller jusqu'à 3 ans avant d'être remboursés. Les gouvernements ne paient pas le plus souvent ce qu'ils doivent aux firmes directement, mais préfèrent accorder un prix plus élevé pour les ventes futures, permettant ainsi un remboursement de la dette. Les autres obstacles comprennent les prix élevés par patient, ce qui a pour conséquence que les cliniques n'ont pas les moyens d'acheter ces médicaments à moins de bénéficier d'un remboursement de l'Etat ou d'un assureur. Une étude d'Eurordis datant de juin sur 12 médicaments orphelins montre que la totalité de ces produits était disponible uniquement au Danemark. La France en avait 11, le Royaume-Uni 8, et les nouveaux membres de l'Union européenne avaient les plus faibles nombres, dont 3 pour la République tchèque et aucun produit en Lituanie et en Lettonie. Une porte-parole de l'EMEA a indiqué à APM Santé que la Commission avait mis en place un groupe de travail pour étudier l'accès des patients aux médicaments orphelins./ni
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21 mai 2005
Médicaments orphelins européens : vers une révision de la durée d’exclusivité commerciale ?
Les médicaments orphelins européens bénéficient d’une exclusivité commerciale de 10 ans après leur mise sur le marché. Cependant, selon l’article 8 de la directive européenne sur les médicaments orphelins (141/2000), cette période d’exclusivité peut être réduite à six ans si, après cinq ans de commercialisation, le médicament orphelin est suffisamment profitable. Alcimed, une société de conseil française, étudie actuellement la relation entre profitabilité et exclusivité commerciale, sur la base des 11 premiers médicaments orphelins ayant une AMM européenne. Elle devrait permettre à la Commission Européenne de développer une méthode d’évaluation de la profitabilité des produits orphelins, d’ici à 2006. Déjà, les industriels craignent une diminution des durées d’exclusivité commerciale. Une décision potentiellement dangereuse car, bien que l’Europe encourage la mise en place de mesures incitatives (crédits d’impôts, financement d’essais cliniques) par les états membres, elles sont peu nombreuses, et c’est principalement l’exclusivité commerciale de 10 ans qui encourage les industriels européens à développer des médicaments orphelins.
Nature Biotechnology ; 22(9) : 1061 ; Septembre 2004
La Commission européenne adopte une proposition de règlement sur les médicaments pédiatriques
Proposition du parlement européen et du conseil relatif aux médicament utilisés en pédiatrie
