Czech Republic Allows Pre-Authorized Use of Zolgensma

Country: CZECH REPUBLIC | Region: EUROPE | Type: Regulation | Keywords: #avexis #chmp #earlyaccess #ema #genetherapy #novartis #pediatrics

PRICENTRIC BRIEF:

  • The Ministry of Health is allowing the implementation of a specific treatment program for pre-authorized use of spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec) by AveXis, a Novartis company
  • Zolgensma will be allowed to treat pediatric patients under the age of two with SMA with a survival motor neuron 1 (SMN1) biallelic mutation regardless of type
  • Adam Vojtech, Minister of Health, said, “We perceive the acute need for this experimental drug by patients, so we have allowed it to be used in the Czech Republic before the final decision of the European Medicines Agency is made”

THE DETAILS

PRAGUE, Czech Republic – The Ministry of Health is allowing the implementation of a specific treatment program for pre-authorized use of spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec) by AveXis, a Novartis company.

Zolgensma will be allowed to treat pediatric patients under the age of two with SMA with a survival motor neuron 1 (SMN1) biallelic mutation regardless of type.

Treatment will be administered in the Neuromuscular Center of the Department of Pediatric Neurology, 2nd Faculty of Medicine, Charles University and Motol University Hospital, explained the Ministry of Health.

While the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHM) adopted a positive option recommending conditional marketing authorization for Zolgensma at its April meeting, the gene therapy has not yet been greenlighted.

In the meantime, the Czech Republic will allow the dispensation, distribution, and use of Zolgensma for patients under the age of two.

Adam Vojtech, Minister of Health, said, “We perceive the acute need for this experimental drug by patients, so we have allowed it to be used in the Czech Republic before the final decision of the European Medicines Agency is made.”

 

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