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New European Pivotal Study Announced by AveXis

09 February 2017

AveXis has announced details of a European pivotal trial of their gene therapy drug for SMA, AVXS-101.

AVXS-101 uses harmless, genetically engineered viruses to increase SMN protein levels, and has recently received “Breakthrough Therapy” status, which could help to speed up the drug approval process (for more information click here). 

This new trial is to be conducted in the European Union (EU) in the second half of 2017, and will replicate the recently announced US pivotal trial (for more information click here).

In the US, a “pivotal trial” is a clinical study designed to provide evidence of drug safety and efficacy that the US Food and Drug Administration (FDA) can then use to determine whether or not to approve a drug for human use.

The European Medicines Agency (EMA), which functions in the EU as the FDA does in the US, provided constructive guidance on the study through the Committee for Medicinal Products for Human Use (CHMP).

Similar to the US trial, the European study will enrol 30 patients with SMA Type 1, and is designed to assess both the safety and effectiveness of AVXS-101. The study is a single-arm trial, meaning that there is no patient group receiving a placebo; rather, the trial will compare the motor milestone achievements of patients receiving AVXS-101 to natural history information from untreated SMA patients.

More specifically, the trial will determine if AVXS-101 improves the ability of patients to sit unaided, and whether it increases the time from birth to a disease-associated event. An event is defined as the time when ventilation support for breathing is required for at least 16 hours a day for 14 consecutive days, or sadly when a patient dies.

AVXS-101 is currently being tested in a Phase I clinical trial, and the latest information coming from the study is that the drug remains well tolerated while patient motor skills are continuing to improve (click here for further information). 


Further Information

AveXis Press Release

AveXis have not yet provided details on how people can get involved in this trial. SMA Support UK will provide an update once this information is available.