Novartis announces plan to give away doses of world’s most expensive drug

Dec. 19, 2019

Next year, Novartis will give away up to 100 doses of its gene therapy for spinal muscular atrophy (SMA), which sells for $2.1 million per patient.

The Swiss drugmaker said that it created the plan specifically to target patients in countries where the treatment, Zolgensma, is not yet approved. Zolgensma was OK’d by the FDA in the U.S. last May and has since raked in $175 million in sales.

SMA impacts about 1 in 10,000 babies, and can lead to severe physical disabilities and death. In some countries awaiting approval for Zolgensma, families have turned to crowd-funding to come up with a way to pay for the treatment, which can completely cure the condition if given before the onset of symptoms.

Biogen’s Spinraza has also been approved to treat SMA and Roche is expected to have a SMA treatment cleared for use in 2020.   

A health advocacy group called TreatSMA said that although it supported Novartis’ efforts to improve access to Zolgensma, it called its program to give away a certain number of doses a “health lottery” for needy patients.

Read the Reuters report.