Baby Pia Denied Compassionate Use: Something ‘Ain’t’ Right in Basel

Novartis just can’t keep out of bad news. They have denied a request from the Belgian federal government to make the most expensive drug in the world (Zolgensma at $2 million) available to a Belgian toddler named Pia who suffers from spinal muscular atrophy, despite crowdfunding success from her parents.
The Family Raises the Funds
Apparently, Pia’s family worked frantically and furiously to save their 9 month old daughter and organized a crowd-funding campaign, raising € 1.9 million needed to afford Zolgensma, a drug yet to receive approval in Europe.
Novartis Refuses to Entertain the Option
Despite the fundraising, the Swiss drug makers denied the 9-month old access to the drug. Belgian Federal Minister Maggie De Block made a plea to the company to “remain human.” The company denied anyway.
The Drug
The world’s most expensive drug, it has been hailed as a breakthrough for the treatment of SMA, a progressive genetic disease leading to muscular failure. Most children diagnosed with the disease die in infancy, reports Ms. Gabriela Galindo in the Brussels Times.
Compassionate Use Access
As the therapy is not approved on the European market, the Belgian minister noted the “largely pub...

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