¿Queréis conocer un poco más sobre la historia de la fenfluramina?

Os contamos hace poco AQUÍ que Fintepla, la marca comercial de Zogenix para la fenfluramina, había sido finalmente aprobada por la agencia americana del medicamento (FDA, por sus siglas en inglés) para la prevención de convulsiones en pacientes mayores de 2 años con síndrome de Dravet.

Stephen Farr, CEO de la empresa propietaria de Fintepla, llamada Zogenix, ha hablado en una entrevista para Dravet Syndrome News, una web especializada en proporcionar información y noticias sobre el síndrome de Dravet.

Farr afirma en dicha entrevista estar “emocionado” y “encantado” por la decisión de la FDA: “El hecho de que hayamos pasado la línea de meta, al menos con una aprobación en los Estados Unidos, nos llena de mucho orgullo, puesto que hemos sido capaces de traducir lo que era un dato prometedor en un número muy pequeño de pacientes en Bélgica en ahora, un producto farmacéutico aprobado del que todos los pacientes con síndrome de Dravet de los Estados Unidos podrían beneficiarse potencialmente”.

¿Cómo se consiguió la aprobación de Fintepla?

La fenfluramina, originalmente desarrollada como un supresor del apetito, demostró ser prometedora en el tratamiento de las convulsiones que caracterizan al síndrome de Dravet, aunque el mecanismo exacto de acción del medicamento aún no está del todo claro.

Los investigadores encontraron pruebas de que la fenfluramina reducía las crisis epilépticas al impedir que las neuronas reutilizaran la serotonina. Sin embargo, investigaciones más recientes sugieren que este fármaco podría también actuar sobre los receptores sigma, un tipo de receptor de membrana celular común en las células nerviosas. En ambos casos, el resultado final es la restauración de una comunicación correcta entre las neuronas.

Lo peculiar de las investigaciones sobre el uso de la fenfluramina para el síndrome de Dravet es que, puesto que la fenfluramina se utilizaba para otros usos terapéuticos, los investigadores ya disponían de datos clínicos antes de iniciar sus estudios en pacientes con Dravet, mientras que la vía lógica en la investigación de los medicamentos es primero estudiar los efectos del tratamiento en organismos modelo en un laboratorio, y luego probarlo en humanos durante los ensayos clínicos.

Fueron los resultados de un estudio belga los que originalmente animaron a Zogenix a investigar la fenfluramina como una terapia comercial. En 2012, los investigadores Berten Ceulemans, Lieven Lagae (actual miembro de nuestro Comité Científico Asesor) y sus colegas demostraron que las personas con síndrome de Dravet experimentaron una marcada reducción de las convulsiones en respuesta a la fenfluramina, incluyendo períodos sin convulsiones mientras estaban siendo tratados con este medicamento.

No fue hasta un año más tarde cuando Zogenix decidió que estos datos eran algo que debían perseguir y se reunió con los investigadores para discutir los resultados.

A pesar de los datos prometedores, la fenfluramina sufrió algunos retrasos en el camino hacia su aprobación. La FDA denegó inicialmente la solicitud de aprobación para Fintepla, alegando, entre otras cosas, que no se habían incluido estudios pre-clínicos (en laboratorio).

Tras discusiones de Zogenix con la FDA, la empresa volvió a presentar su solicitud a finales de 2019, y Fintepla fue finalmente aprobada en EEUU el pasado 25 de junio.

¿Cuánto cuesta Fintepla?

El coste promedio de Fintepla en EEUU se estima en 96.000 dólares al año. Farr comenta que el precio “se basa realmente en el hecho de que se ha demostrado una enorme eficacia en el síndrome de Dravet”. El precio final del tratamiento individual variará un poco, ya que la medicación se dosifica según el peso del paciente.

“Estamos poniendo en marcha sistemas y servicios de apoyo que asegurarán que el precio para los pacientes sea muy pequeño, o incluso nulo, de modo que el coste sea para los pagadores (aseguradoras, agencias gubernamentales, etc.) y no para el paciente”, afirma Farr.

Zogenix establece programas de asistencia al paciente:

Puesto que Fintepla conlleva el riesgo de desarrollar una enfermedad cardíaca valvular e hipertensión arterial pulmonar, Zogenix ha establecido algo llamado ‘Programa de Evaluación y Mitigación de Riesgos’ (REMS, por sus siglas en inglés). Este programa tiene como objetivo asegurar que el medicamento sea únicamente prescrito y distribuido por proveedores certificados y que los pacientes reciban una monitorización cardíaca adecuada. A pesar de este riesgo, Farr enfatiza que no ha habido casos de tales complicaciones durante las pruebas.

“El elemento más importante para asegurar un uso seguro es la realización de ecocardiogramas. Hemos realizado más de 6.000 ecocardiogramas en niños, por lo que tenemos probablemente la mayor base de datos de ecocardiogramas en niños de todo el mundo”.

Mientras que Farr dice que no ha habido evidencia de enfermedad cardíaca en los ensayos clínicos de fase 3 (aquellos en los que se estudia si el nuevo medicamento es mejor que otros ya existentes), la asociación entre estas condiciones y los medicamentos serotoninérgicos como Fintepla hace que sea importante la monitorización de pacientes a través del programa REMS una vez que el producto esté disponible comercialmente. Farr añadió que también han trabajado para poner en marcha un sistema por el cual los ecocardiogramas se puedan realizar en los centros más cercanos a donde viven los pacientes, en lugar de en los hospitales principales.

Todos los requerimientos del REMS, como la programación de ecocardiogramas, serán coordinados a través de un programa llamado ‘Zogenix Central’, que servirá como centro logístico a través del cual se manejará el cuidado y la información de los pacientes. “Asegurará que los pacientes y los cuidadores sean informados sobre el uso seguro de Fintepla y sobre las posibilidades de reembolso de su coste”, dijo Farr.

A través de Zogenix Central, las farmacias certificadas enviarán el medicamento directamente a la casa del cuidador. Este programa está diseñado, dice Farr, para hacer que cualquier obstáculo relacionado con la obtención de Fintepla sea invisible para los pacientes y cuidadores.

¿Se puede usar Fintepla con otros medicamentos?

Fintepla puede prescribirse junto con otros medicamentos con pocas restricciones, según Farr, pero puede ser necesario ajustar la dosis si se toma en combinación con otras terapias. Por ejemplo, si Fintepla se prescribe con Diacomit (estiripentol), podría ser necesaria la reducción de dosis de este último. “Por el contrario”, dice Farr, “no hay reducción de dosis si un médico quiere probar el cannabidiol (CBD) en combinación con Fintepla”.

El uso potencial de Fintepla junto con el CBD podría ser una consideración importante a la luz de la aprobación por la FDA en 2018, y por la Agencia Española del Medicamento y Productos Sanitarios (AEMPS) de Epidyolex, una solución de CBD desarrollada por GW Pharmaceuticals para el tratamiento de las convulsiones en el síndrome de Dravet. Con este fin, un ensayo clínico de fase 1/2 (NCT03467113) está evaluando actualmente la seguridad de la fenfluramina en combinación con CBD para pacientes con síndrome de Dravet o Lennox-Gastaut.

Además, “la fenfluramina no afecta a los niveles sanguíneos de ninguno de los otros medicamentos antiepilépticos”, dice Farr, lo cual es importante señalar ya que las personas con Dravet a menudo son tratadas con múltiples terapias.

Fintepla también está actualmente bajo revisión para su aprobación en la Unión Europea, con una decisión anticipada para finales de año, según Farr.

La información aquí proporcionada ha sido extraída del artículo de Forest Ray AQUÍ.

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TERMS & CONDITIONS

The Dravet Syndrome Conference 2023 is organised by Dravet Syndrome Foundation Spain, whose registered office is located at Calle Doctor Fleming, 30, 28036 Madrid, Spain.

The conference shall take place at the Official College of Nursing of Madrid, at Avenida de Menéndez Pelayo, 93, 28007 Madrid, Spain, on Thursday 23rd of March 2023. Attendance to this conference is free of charge, but mandatory for organisational reasons.

Participants must register online at www.dravetconference.com. Upon registration, each participant will receive an electronic confirmation by email. On-site registration will not be be possible. If a participant has not received the official email registration confirmation within 3 days, they must contact the conference secretariat at [email protected].

The conference registration includes: Attendance to all sessions, oral and visual presentations, all coffee and lunch breaks, and conference documentation with all information about the event. The registration does not include travel and accommodation costs or any other personal expenses.

Admittance to the conference without registration cannot be granted.

Conference Attendance
Upon arrival at the Official College of Nursing of Madrid the day of the event, all participants have to check-in at the onsite Conference Registration Desk. The desk will open on 23rd of March at 8:30 a.m. Upon registration onsite, the conference team will provide participants with information about the event and a badge, which should be worn visibly at all times. The personalised badge authorises participants to attend all conference sessions, and lunch and coffee breaks.

Cancellation Policy
Dravet Syndrome Foundation Spain reserves the right to cancel any registration forthwith and without liability on its part in the event of any damage to, or destruction of the venue by fire, shortage of labour, strikes, industrial unrest, public health measures, or any other cause beyond the control of the Dravet Syndrome Foundation Spain, which shall prevent it from performing its obligations. In these circumstances, every effort will be made to find an alternative format/venue or propose a new date for the conference. Should the conference be cancelled for any reason, airfares, hotels, or other costs incurred by the participants are not reimbursed by the Dravet Syndrome Foundation Spain.

Any participant is allowed to cancel his/her registration but must notify this to the conference organisers by e-mail at [email protected] within a minimum of 10 days prior the event. Failure to attend the event without prior written notification by email may incur a no-show fee, which shall cover individual organizational, material and catering costs.

Name Change
All registrations are only valid for the respective person mentioned in the form. A registered participant unable to attend the conference may nominate (free of charge) a substitute participant by notifying the conference organisers by email at [email protected] before 16th of March 2023. The procedure allows giving the last name, first name, and email address of the new participant.

Personal Data
Upon registration, the participant authorises Dravet Syndrome Foundation Spain to use their personal data stated above to inform the participant of any further conferences, events, or information concerning the conference and Dravet Syndrome Foundation Spain’s activities. The conference booklet online will only contain authors, titles, and affiliations of submitted abstracts, but no full abstract texts. The conference printed booklet will contain full abstract texts from all participants. Conference certificates will be sent to the participants as a PDF document upon request at [email protected]. Only the name & affiliation will be stated in the certificate, not the email addresses of the participants.

Information is collected by an automated computer system that manages registrations. Security and confidentiality are guaranteed by state-of-the-art technical tools. However, Dravet Syndrome Foundation Spain declines any responsibility concerning data security transmitted through the internet.

Photography
The conference is held in public; therefore, we do not ban participants, exhibitors, partners and other companies from taking photographs of conference activities and participants, and share it via social media. By participating in the conference, you approve that general pictures will be taken and used for our Social Media output, our websites and other communication purposes. You hereby waive any right to oppose, and expressly authorise the organiser to make them available on the Internet or as it may seem fit. Author’s presentation slides shall not be photographed by any conference participant. 

Disclaimer
The conference organiser cannot be held responsible for any loss, injury or damage to any property, whatever the cause may be. Should, for any reason outside the organiser’s control (e.g., political or economic circumstances or a case of ‘force majeure’), the venue or speakers change, or the event be cancelled, the organiser will endeavour to reschedule, but shall not be held responsible for any costs, damages or expenses incurred by the participants. If for any reason the organiser decides to make material changes to this conference, the organiser is not responsible for airfares, hotels or other costs incurred by the participants. The participant takes part in the conference, all tours, and trips at his own risk.

Copyright
The participant is allowed to use the conference documentation exclusively for his/her personal needs.

TERMS & CONDITIONS

The European Dravet Syndrome Advanced Therapies (EDSAT) Meeting 2023 is organised by Dravet Syndrome Foundation Spain, whose registered office is located at Calle Doctor Fleming, 30, 28036 Madrid, Spain.

The conference shall take place at the Official College of Nursing of Madrid, at Avenida de Menéndez Pelayo, 93, 28007 Madrid, Spain, on Friday 24th of March 2023. Attendance to this conference is free of charge, but mandatory for organisational reasons.

Participants must register online at www.dravetconference.com. Upon registration, each participant will receive an electronic confirmation by email. On-site registration will not be be possible. If a participant has not received the official email registration confirmation within 3 days, they must contact the conference secretariat at [email protected].

The conference registration includes: Attendance to all sessions, oral and visual presentations, all coffee and lunch breaks, and conference documentation with all information about the event. The registration does not include travel and accommodation costs or any other personal expenses.

Admittance to the conference without registration cannot be granted.

Conference Attendance
Upon arrival at the Official College of Nursing of Madrid the day of the event, all participants have to check-in at the onsite Conference Registration Desk. The desk will open on 24th of March at 8:30 a.m. Upon registration onsite, the conference team will provide participants with information about the event and a badge, which should be worn visibly at all times. The personalised badge authorises participants to attend all conference sessions, and lunch and coffee breaks.

Cancellation Policy
Dravet Syndrome Foundation Spain reserves the right to cancel any registration forthwith and without liability on its part in the event of any damage to, or destruction of the venue by fire, shortage of labour, strikes, industrial unrest, public health measures, or any other cause beyond the control of the Dravet Syndrome Foundation Spain, which shall prevent it from performing its obligations. In these circumstances, every effort will be made to find an alternative format/venue or propose a new date for the conference. Should the conference be cancelled for any reason, airfares, hotels, or other costs incurred by the participants are not reimbursed by the Dravet Syndrome Foundation Spain.

Any participant is allowed to cancel his/her registration but must notify this to the conference organisers by e-mail at [email protected] within a minimum of 10 days prior the event. Failure to attend the event without prior written notification
by email may incur a no-show fee, which shall cover individual organizational, material and catering costs.

Name Change
All registrations are only valid for the respective person mentioned in the form. A registered participant unable to attend the conference may nominate (free of charge) a substitute participant by notifying the conference organisers by email at [email protected] before 17th of March 2023. The procedure allows giving the last name, first name, and email address of the new participant.

Personal Data
Upon registration, the participant authorises Dravet Syndrome Foundation Spain to use their personal data stated above to inform the participant of any further conferences, events, or information concerning the conference and Dravet Syndrome Foundation Spain’s activities. The conference booklet online will only contain authors, titles, and affiliations of submitted abstracts, but no full abstract texts. The conference printed booklet will contain full abstract texts from all participants. Conference certificates will be sent to the participants as a PDF document upon request at [email protected]. Only the name & affiliation will be stated in the certificate, not the email addresses of the participants.

Information is collected by an automated computer system that manages registrations. Security and confidentiality are guaranteed by state-of-the-art technical tools. However, Dravet Syndrome Foundation Spain declines any responsibility concerning data security transmitted through the internet.

Photography
The conference is held in public; therefore, we do not ban participants, exhibitors, partners and other companies from taking photographs of conference activities and participants, and share it via social media. By participating in the conference, you approve that general pictures will be taken and used for our Social Media output, our websites and other communication purposes. You hereby waive any right to oppose, and expressly authorise the organiser to make them available on the Internet or as it may seem fit. Author’s presentation slides shall not be photographed by any conference participant.

Disclaimer
The conference organiser cannot be held responsible for any loss, injury or damage to any property, whatever the cause may be. Should, for any reason outside the organiser’s control (e.g., political or economic circumstances or a case of ‘force majeure’), the venue or speakers change, or the event be cancelled, the organiser will endeavour to reschedule, but shall not be held responsible for any costs, damages or expenses incurred by the participants. If for any reason the organiser decides to make material changes to this conference, the organiser is not responsible for airfares, hotels or other costs incurred by the participants. The participant takes part in the conference, all tours, and trips at his own risk.

Copyright
The participant is allowed to use the conference documentation exclusively for his/her personal needs.

TERMS & CONDITIONS

The European Dravet Syndrome Advanced Therapies (EDSAT) Meeting 2022 is organised by Dravet Syndrome Foundation Spain, whose registered office is located at Calle Doctor Fleming, 30, 28036 Madrid, Spain.

The conference shall take place at the Illustrious Official College of Physicians of Madrid, at Calle de Santa Isabel, 51, 28012 Madrid, Spain, on Friday 1st of April 2022. Attendance to this conference is free of charge, but mandatory for organisational reasons. Participants must register online at www.dravetconference.com. Upon registration, each participant will receive an electronic confirmation by email. On-site registration will not be be possible. If a participant has not received the official email registration confirmation within 3 days, they must contact the conference secretariat at [email protected].

The conference registration includes: Attendance to all sessions, oral and visual presentations, all coffee and lunch breaks, and conference documentation with all information about the event. The registration does not comprise travelling and accommodation costs or any other personal expenses.

Admittance to the workshop without registration cannot be granted.

Conference Attendance
Upon arrival at the Illustrious Official College of Physicians of Madrid the day of the event, all participants have to register at the onsite Conference Registration Desk. The desk will open on 1st of April at 8:30 a.m. Upon registration onsite, the conference team will provide participants with information about the event and a badge, which should be worn visibly at all times. The personalised badge authorises participants to attend all conference sessions, and lunch and coffee breaks.

Cancellation Policy
Dravet Syndrome Foundation Spain reserves the right to cancel any registration forthwith and without liability on its part in the event of any damage to, or destruction of the venue by fire, shortage of labour, strikes, industrial unrest, public health measures, or any other cause beyond the control of the Dravet Syndrome Foundation Spain, which shall prevent it from performing its obligations. In these circumstances, every effort will be made to find an alternative format/venue or propose a new date for the conference. Should the conference be cancelled for any reason, airfares, hotels, or other costs incurred by the participants are not reimbursed by the Dravet Syndrome Foundation Spain.

Any participant is allowed to cancel his/her registration but must notify this to the conference organisers by e-mail at [email protected] within a minimum of 10 days
prior the event. Failure to attend the event without prior written notification
by email may incur a no-show fee, which shall cover individual organizational,
material and catering costs.

Name Change
All registrations are only valid for the respective person mentioned in the form. A registered participant unable to attend the conference may nominate (free of charge) a substitute participant by notifying the conference organisers by email at [email protected] before 24th of March 2022. The procedure allows giving the last name, first name, and email address of the new participant.

Personal Data
Upon registration, the participant authorises Dravet Syndrome Foundation Spain to use their personal data stated above to inform the participant of any further conferences, events, or information concerning the conference and Dravet Syndrome Foundation Spain’s activities. The conference booklet online will only contain authors, titles, and affiliations of submitted abstracts, but no full abstract texts. The conference printed booklet will contain full abstract texts from all participants. Conference certificates will be sent to the participants as a PDF document upon request at [email protected]. Only the name & affiliation will be stated in the certificate, not the email addresses of the participants.

Information is collected by an automated computer system that manages registrations. Security and confidentiality are guaranteed by state-of-the-art technical tools. However, Dravet Syndrome Foundation Spain declines any responsibility concerning data security transmitted through the internet.

Photography
The conference is held in public; therefore, we do not ban participants, exhibitors, partners and other companies from taking photographs of conference activities and participants, and share it via social media. By participating in the conference, you approve that general pictures will be taken and used for our Social Media output, our websites and other communication purposes. You hereby waive any right to oppose, and expressly authorise the organiser to make them available on the Internet or as it may seem fit. Author’s presentation slides shall not be photographed by any conference participant.

Disclaimer
The conference organiser cannot be held responsible for any loss, injury or damage to any property, whatever the cause may be. Should, for any reason outside the organiser’s control (e.g., political or economic circumstances or a case of ‘force majeure’), the venue or speakers change, or the event be cancelled, the organiser will endeavour to reschedule, but shall not be held responsible for any costs, damages or expenses incurred by the participants. If for any reason the organiser decides to make material changes to this conference, the organiser is not responsible for airfares, hotels or other costs incurred by the participants. The participant takes part in the conference, all tours, and trips at his own risk.

Copyright
The participant is allowed to use the conference documentation exclusively for his/her personal needs.

TERMS & CONDITIONS

The Dravet Syndrome Conference 2022 is organised by Dravet Syndrome Foundation Spain, whose registered office is located at Calle Doctor Fleming, 30, 28036 Madrid, Spain.

The conference shall take place at the Illustrious Official College of Physicians of Madrid, at Calle de Santa Isabel, 51, 28012 Madrid, Spain, on Thursday 31st of March 2022. Attendance to this conference is free of charge, but mandatory for organisational reasons. Participants must register online at www.dravetconference.com. Upon registration, each participant will receive an electronic confirmation by email. On-site registration will not be be possible. If a participant has not received the official email registration confirmation within 3 days, they must contact the conference secretariat at [email protected].

The conference registration includes: Attendance to all sessions, oral and visual presentations, all coffee and lunch breaks, and conference documentation with all information about the event. The registration does not include travel and accommodation costs or any other personal expenses.

Admittance to the conference without registration cannot be granted.

Conference Attendance
Upon arrival at the Illustrious Official College of Physicians of Madrid the day of the event, all participants have to register at the onsite Conference Registration Desk. The desk will open on 31st of March at 8:30 a.m. Upon registration onsite, the conference team will provide participants with information about the event and a badge, which should be worn visibly at all times. The personalised badge authorises participants to attend all conference sessions, and lunch and coffee breaks. 

Cancellation Policy
Dravet Syndrome Foundation Spain reserves the right to cancel any registration forthwith and without liability on its part in the event of any damage to, or destruction of the venue by fire, shortage of labour, strikes, industrial unrest, public health measures, or any other cause beyond the control of the Dravet Syndrome Foundation Spain, which shall prevent it from performing its obligations. In these circumstances, every effort will be made to find an alternative format/venue or propose a new date for the conference. Should the conference be cancelled for any reason, airfares, hotels, or other costs incurred by the participants are not reimbursed by the Dravet Syndrome Foundation Spain.

Any participant is allowed to cancel his/her registration but must notify this to the conference organisers by e-mail at [email protected] within a minimum of 10 days prior the event. Failure to attend the event without prior written notification by email may incur a no-show fee, which shall cover individual organizational, material and catering costs.

Name Change
All registrations are only valid for the respective person mentioned in the form. A registered participant unable to attend the conference may nominate (free of charge) a substitute participant by notifying the conference organisers by email at [email protected] before 24th of March 2022. The procedure allows giving the last name, first name, and email address of the new participant.

Personal Data
Upon registration, the participant authorises Dravet Syndrome Foundation Spain to use their personal data stated above to inform the participant of any further conferences, events, or information concerning the conference and Dravet Syndrome Foundation Spain’s activities. The conference booklet online will only contain authors, titles, and affiliations of submitted abstracts, but no full abstract texts. The conference printed booklet will contain full abstract texts from all participants. Conference certificates will be sent to the participants as a PDF document upon request at [email protected]. Only the name & affiliation will be stated in the certificate, not the email addresses of the participants.

Information is collected by an automated computer system that manages registrations. Security and confidentiality are guaranteed by state-of-the-art technical tools. However, Dravet Syndrome Foundation Spain declines any responsibility concerning data security transmitted through the internet.

Photography
The conference is held in public; therefore, we do not ban participants, exhibitors, partners and other companies from taking photographs of conference activities and participants, and share it via social media. By participating in the conference, you approve that general pictures will be taken and used for our Social Media output, our websites and other communication purposes. You hereby waive any right to oppose, and expressly authorise the organiser to make them available on the Internet or as it may seem fit. Author’s presentation slides shall not be photographed by any conference participant. 

Disclaimer
The conference organiser cannot be held responsible for any loss, injury or damage to any property, whatever the cause may be. Should, for any reason outside the organiser’s control (e.g., political or economic circumstances or a case of ‘force majeure’), the venue or speakers change, or the event be cancelled, the organiser will endeavour to reschedule, but shall not be held responsible for any costs, damages or expenses incurred by the participants. If for any reason the organiser decides to make material changes to this conference, the organiser is not responsible for airfares, hotels or other costs incurred by the participants. The participant takes part in the conference, all tours, and trips at his own risk.

Copyright
The participant is allowed to use the conference documentation exclusively for his/her personal needs.