Swiss drugmaker Novartis won a key European recommendation for the company’s gene therapy Zolgensma against spinal muscular atrophy (SMA), clearing a hurdle for $2.1 million per patient treatment for approval in Europe within months.
Genentech’s Risdiplam showed significant improvement in motor function in people aged 2-25 who have been diagnosed with Type 2 or 3 spinal muscular atrophy.
Roche company Genentech announced positive top-line results from the pivotal Part 2 of the FIREFISH study looking at risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA).
Tokyo-based Astellas Pharma announced plans to acquire San Francisco-based Audentes Therapeutics for $60 per share in cash, representing a total equity value of about $3 billion.
Vertex Pharmaceuticals will acquire privately held Exonics Therapeutics for an upfront payment of $245 million and potential milestone payments of up to $750 million.
England’s National Health Service reached a deal to pay for U.S. drugmaker Biogen Inc.’s expensive spinal muscle atrophy treatment Spinraza.
Scholar Rock – a biotechnology company focused on discovering and developing drugs that selectively target growth factors in the disease microenvironment – announced the publication of “Blocking extracellular activation of myostatin as a strategy for treating muscle wasting,” in Scientific Reports, the peer-reviewed, open-access journal published by Nature.
Biogen and Ionis Pharmaceuticals announced that they entered into a new collaboration agreement to identify new antisense oligonucleotide drug candidates for the treatment of spinal muscular atrophy (SMA).
