Skip to NavigationSkip to content

sma

Novartis' $2.15m SMA drug Zolgensma becomes most expensive FDA-approved therapy

The FDA has approved Novartis subsidiary AveXis’ $2.15 million gene therapy Zolgensma for children with spinal muscular atrophy (SMA).

The treatment has become the first FDA-approved gene therapy for children with SMA.

The drug has been approved for patients living with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene, under the age of two.

Zolgensma is designed to address the genetic root cause of SMA in providing a functional copy of the SMN gene. The treatment is delivered intravenously with a one-time infusion.

NHS England agree to pay for Biogen's $750,000 a year SMA treatment Spinraza

NHS England has agreed to pay an undisclosed price for Biogen’s spinal muscle atrophy (SMA) treatment Spinraza.

England’s National Health Service said it had reached a ‘managed access agreement’ under which it will fund the treatment for a limited period of time, while collecting data on the drug’s effectiveness.

Spinraza, which is administered every four months via spinal injections, has a US list price of $750,000 for the first year and $375,000 for every year after.

Protesters in Manchester call on NICE to approve Biogen's $750,000 drug Spinraza

Image Credit: Juliux | Manchester Town Hall

Protesters in Manchester have called on the National Institute of Health and Care Excellence (NICE), to overturn their rejection of the Spinal Muscular Atrophy (SMA) treatment Spinraza, after the public body chose not to approve the orphan drug for use on the NHS due “to uncertainties over its long-term effectiveness and its extremely high cost.”

Roche buys French biotech for €470 million

Roche image

Roche is buying France-based biotech Trophos in a deal worth up to €470 million in order to gain access to its promising Phase II drug olesoxime.

Trophos is developing olesoxime (TRO19622) for spinal muscular atrophy (SMA) – which is a rare and debilitating genetic neuromuscular disease that is most commonly diagnosed in children.

Mission Statement
Pharmafile.com is a leading portal for the pharmaceutical industry, providing industry professionals with pharma news, pharma events, pharma service company listings and pharma jobs,
Site content is produced by our editorial team exclusively for Pharmafile.com and our industry newspaper Pharmafocus. Service company profiles and listings are taken from our pharmaceutical industry directory, Pharmafile, and presented in a unique Find and Compare format to ensure the most relevant matches