Spinal muscular atrophy

“Think of it as an installment plan that’s then tied to how well the therapy works,” Mark Trusheim, strategic director of the MIT Center for Biomedical Innovation’s NEWDIGS program, told Business Insider. “This would be a car loan but you’ve still got to see if the car is going to work.”

Using a calculation, ICER believes the Novartis gene therapy for Spinal Muscular Atrophy will be more cost effective than Biogen’s Spinraza.

Novartis’ gene therapy treatment for spinal muscular atrophy could see approval as early as May of this year after the U.S. Food and Drug Administration granted AVXS-101, now dubbed Zolgensma, priority review.

PTC Therapeutics announced interim data from Part 1 of its open-label clinical trials, FIREFISH and SUNFISH, of risdiplam for Type 1, 2 and 3 spinal muscular atrophy in collaboration with Genentech.

PTC Therapeutics presented updated interim clinical data from Part 1 of its FIREFISH clinical trial of risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy.

Cytokinetics released data from its Phase II clinical trial of reldesemtiv in patients with spinal muscular atrophy (SMA).

Biogen saw a strong first quarter with 11 percent growth in revenues to $3.1 billion, which were spurred by gains in spinal muscular atrophy (SMA) treatment Spinraza and multiple sclerosis treatment Tecfidera.

The company believes the Translarna data is compelling and pointed to the success the drug is having in Europe.