Roche’s Evrysdi continues to improve motor function and survival in babies with Type 1 Spinal Muscular Atrophy (SMA) - Seite 2

Ninety-three percent of infants (38/41) were alive after 24 months of treatment. Eighty-three percent of patients (34/41) were alive and free from permanent ventilation after 24 months, an improvement compared to the natural course of the disease. There were no new deaths between months 12 and 24. Without treatment, the median age of death or permanent ventilation is 13.5 months. In addition, fewer hospitalisations were observed during the second year of treatment with Evrysdi compared with the natural course of the disease, with 34% of infants (14/41) not requiring hospitalisation during 24 months of treatment. Additional findings suggested that Evrysdi continued to improve measures of the Hammersmith Infant Neurological Examination 2 (HINE-2) at month 24 vs. month 12, including being able to hold their head upright (63% vs. 44%), roll from supine to prone (44% vs. 10%), stand with support (15% vs. 5%) and walk** (4% vs. 2%). Continued improvements were also observed in CHOP-INTEND*** total score, with a larger percentage of patients achieving a score of at least 40 by month 24 (76%; 31/41) than month 12 (56%; 23/41). In the natural course of the disease, children with Type 1 SMA rarely reach a CHOP-INTEND total score of 40 points.

“These data highlight the real-world impact of this transformative medicine in babies with the most severe form of SMA. For example, all infants alive after 24 months of treatment were able to swallow which can help them to feed orally rather than through a tube,” said Levi Garraway, M.D., Ph. D., Chief Medical Officer and Head of Global Product Development. “These results increase our understanding of how this first-of-its-kind treatment can extend the lives of babies with Type 1 SMA, providing much needed hope for their families.”

The adverse events and serious adverse events observed were consistent with previous studies. The most common adverse events were upper respiratory tract infection (54%), pneumonia (46%), pyrexia (44%), constipation (29%), nasopharyngitis (17%), bronchitis (15%), diarrhea (15%) and rhinitis (12%). The incidence of serious pneumonia declined by approximately 3-fold between the first and second 12-month periods of FIREFISH Part 2. The most common serious adverse events were pneumonia (39%) and respiratory distress (7%). There were no drug-related adverse events leading to withdrawal or treatment discontinuation.