Topic: spinal muscular atrophy
The FDA halted enrollment in one arm of the phase 1/2 Strong trial, which is testing Novartis' gene therapy Zolgensma as a spinal injection.
Zolgensma hauled in $160 million in its first full quarter on the market, more than 60% above the Street's expectations.
Novartis is touting data showing dramatic improvements after treatment with Zolgensma for SMA patients between ages 2 and 5.
JPMorgan discovered concerns about the efficacy of Novartis' Zolgensma that could make it difficult for the SMA gene therapy to meet expectations.
Alongside long-term data showing patients were still benefiting from Zolgensma, Novartis said a trial patient's death wasn't related to the therapy.
Biogen plans to test its SMA drug Spinraza at a higher-than-approved dose as Novartis' rival gene therapy Zolgensma picks up traction.
Biogen execs were forced to explain why their fast-launching SMA drug Spinraza stopped in its tracks last quarter.
Novartis is struggling to gain insurance coverage for Zolgensma, signaling a tough road ahead for gene therapy, Bernstein analysts said.
All presymptomatic SMA patients in a trial of Biogen's Spinraza are alive and can sit independently. And 88% of patients can walk independently.
Spinal muscular atrophy gene therapy Zolgensma has nabbed an FDA nod to challenge Biogen's Spinraza.