[ad_1] Swiss drugmaker Novartis recently launched a lottery-style program to allocate free doses of Zolgensma, its groundbreaking treatment for children with spinal muscular atrophy (SMA), to patients in countries that haven’t approved the drug. But with a $2.1 million price tag, Zolgensma is inaccessible to some patients even where it has been approved. The prohibitive …
Targets the genetic root cause of SMA with a one-time-only dose. ZOLGENSMA ® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA).
Dec 20, 2019 · Novartis will give away 100 doses of its most expensive drug, Zolgensma. Some patient advocates worry that a “health lottery” system is not an appropriate way to treat a serious condition like spinal muscular atrophy and that it could neglect some patients.
Dec 19, 2019 · Yes, but advocates worry about Novartis' lottery-style giveaway. Novartis' Zolgensma is the world's most expensive at $2.1 million per one-time use in the U.S. It's not yet approved elsewhere.
Babies hoping to get Zolgensma as soon as possible. Right now, their only chance is Novartis’ global managed access program (aka “the lottery”), which every two weeks randomly selects two babies from a worldwide pool of applicants to receive a compassionate treatment.
Novartis plans to give away up to 100 doses annually of Zolgensma, its $2.1 million spinal muscular atrophy treatment, under a new managed-access program, according to TreatSMA.
Aug 06, 2019 · This photo provided by Novartis shows their gene therapy medicine Zolgensma. U.S. regulators want to know why Novartis didn’t disclose a problem with testing data until after they approved the ...
Dec 20, 2019 · The company announced this week that starting next month, its AveXis unit will begin distributing doses of Zolgensma, a one-time gene treatment for spinal muscular atrophy, also known as SMA. The disease affects about 1 in 10,000 births, and which results in death or the need for permanent ventilation by the age of two in 90 percent of cases.
Novartis plans to giveaway 50 doses of the world’s costliest single-dose treatment, Zolgensma, for babies under 2 years old, with up to 100 doses being distributed throughout 2020. Zolgensma is designed to treat spinal muscular atrophy (SMA) and costs $2.1 million-per-patient. It had sales of $175 million in September 2019.