Informe favorable del CHMP de la EMA sobre Zolgensma y EPAR

31/5/20

Zolgensma ya tiene EPAR con condiciones desde el día 27 de mayo de 2020 

4/4/20

En su reunión del 26 Marzo de 2020, el  Comité de  Productos Medicinales para uso Humano  (CHMP) adoptó una opinión positiva, recomendando la emisión de la autorización de comercialización de Zolgensma, que va dirigido al tratamiento de niños y jóvenes con Atrofia Muscular Espinal (SMA  en inglés) del que nos hemos ocupado en varias ocasiones en este blog:

• Zolgensma, de Novartis, posible para mayo en la FDA
• Informe final de ICER sobre Zolgensma
• Aprobado Zolgensma por la FDA. Su precio USD 2.1M 
• T Cigna asume el pago de Luxturna y Zolgensma
• Resultados económicos de las CART hasta Q3 del 2019, y sobre Zolgensma y sus circunstancias.

Las indicaciones serán: 

Zolgensma is indicated for the treatment of: 

- patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or

- patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.

The applicant for Zolgensma is AveXis EU Limited.

Las bases de la autorización son:

The clinical trial providing the main body of data for the assessment of Zolgensma was conducted in 22 patients who were less than six months of age at the time of the gene replacement therapy with Zolgensma. The trial assessed the percentage of patients who had improvement in their survival (i.e. without the need to be permanently on a ventilator) and motor milestones, such as head control, crawling, sitting, standing and walking (with or without assistance).
The survival of patients treated with Zolgensma exceeded what can be expected from untreated patients with severe SMA. Out of 22 patients enrolled in the trial, 20 patients (91%) were alive and did not need permanent ventilatory support at 14 months of age. The experience with this disease shows that at 14 months of age only 25% of patients are still alive. These patients also achieved motor milestones, which are usually not achieved in the natural history of the disease. 14 patients (64%) reached the milestone of independent sitting before 18 months of age. One patient (4%) reached the milestone of walking unassisted before reaching 16 months of age. Patients with less motor deterioration appeared to benefit the most from the treatment with Zolgensma.

El paso siguiente será la publicación  del EPAR.

Han pasado varios meses desde la ultima autorización de comercialización de un MTA: Autorizado un nuevo ATMP: ZYNTEGLO en junio de 2019.