Topic: spinal muscular atrophy
Novartis plans to give away doses of its $2.1 million gene therapy Zolgensma, but critics are worried about its 'too crude' lottery approach.
Roche's risdiplam, a potential 2020 entrant in spinal muscular atrophy, scored a FDA priority review on Monday.
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.