Biogen Inc. said a phase 3 trial of Spinraza demonstrated improved motor function in infant and children with spinal muscle atrophy, or SMA, a rare neuromuscular disorder.
Results from the Endear study showed the drug demonstrated greater benefit and improvement in motor function outcomes in infants when compared to those who were untreated.
The drug was better in both, infants whose disease duration was either less than or equal to 12 weeks and whose disease duration was greater than 12 weeks. Additionally, infants in the former group experienced a significant benefit in event-free survival.
In a separate study, which is still ongoing, the drug is said to have shown a larger proportion of infants and children responding to Spinraza compared to those who were untreated.
The interim analysis from the phase 2 Embrace study also supports the dosing regimen of four loading doses in the first two months, followed by the administration of Spinraza every four months thereafter.
"These studies contribute to a growing body of evidence that Spinraza can make a meaningful difference in the lives of people with SMA regardless of their age or stage of the disease. Across studies, we continue to see evidence that earlier initiation of treatment with SPINRAZA can lead to improved clinical and functional outcomes," Biogen's chief medical officer and executive vice president Alfred Sandrock said in a statement.
Spinraza, which is already approved in Brazil, Japan, Canada, the U.S. and the EU, generated $203 million in second-quarter revenue, beating consensus expectations of roughly $70 million.
However, sales are likely to slow as patients move from a $500,000 initial eight-week round of treatment to maintenance therapy, which is about $250,000 over 32 weeks.
Biogen licensed Spinraza from Ionis Pharmaceuticals Inc. and is looking to introduce the drug in additional geographies.