ZURICH, Jan 23 (Reuters) - Roche’s risdiplam met the primary endpoint in a pivotal clinical trial in infants with type 1 spinal muscular atrophy, the Swiss drugmaker said on Thursday.
The treatment demonstrated statistically significant and medically meaningful motor milestone improvement in infants with Type 1 SMA, it said in a statement. No treatment-related safety findings leading to withdrawal were seen in any risdiplam trial to date.
Roche said this month it plans to price risdiplam aggressively to challenge two of the world’s most expensive medicines, Biogen’s Spinraza and Novartis’s gene therapy Zolgensma. (Reporting by Michael Shields)