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Aprobado Zolgensma por la FDA. Su precio USD 2.1M

El pasado día 24 de mayo de 2019 aprobó la FDA la comercialización de ZOLGENSMA tal como parecía que iba a ser.
Copiamos de la web de la FDA:
STN: 125694
Proper Name: onasemnogene abeparvovec-xioi
Trade Name: ZOLGENSMA®
Manufacturer: AveXis, Inc 
Indication: For the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
Product Information Package Insert-ZOLGENSMA
Supporting Documents May 24, 2019 Approval Letter - ZOLGENSMA

En la página web de Novartis habla del precio:
The current 10-year cost of chronic therapy, which is given over the patient's lifetime, can often exceed USD 4 million in just the first 10 years of a young child's life.[1] In addition, that therapy stops working if treatment is stopped.[2] Zolgensma is expected to save costs in the healthcare system compared to chronic treatment for the treatment and care of SMA.[3] The wholesale acquisition cost of Zolgensma of USD 2.125 million is:
  •  50% of the 10-year cost of current chronic SMA treatment (estimated at USD 4.1 million)[1] 
  • 50% below 10-year treatment costs for genetic pediatric ultra-rare diseases (estimated at USD 4.4 million to USD 5.7 million)[4]* 
  • 50% below the ICER ultra-rare disease cost-effectiveness threshold; Zolgensma pricing places it at approximately USD 250,000 per quality-adjusted life-year (QALY)[5]

y de acuerdos con pagadores:
AveXis has partnered with Accredo® to offer a pay-over-time option of up to 5 years to help ease possible short-term budget constraints, especially for states, small payers and self-insured employers. In addition, CuraScript SD® has been selected as the sole specialty distributor given its rare disease experience, including gene and cell therapies.

Comentarios

  1. Gracias por esta publicación, ya está aquí la posible solución para esta terrible enfermedad. Ojalá

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  2. Será cuestión de ver las publicaciones del seguimiento y que a lo largo del tiempo continúen los buenos resultados - ya hay niños con más de 4 años desde el tratamiento- y ver que no aparecen efectos no deseados.
    Se supone que la mejora de las técnicas de elaboración de los AAV, que tú conoces, harán que el precio no sea tan astronómico.

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