EMA approves new measure for DMD drug trials that can replace the Six Minute Walking Test
It has been announced that the European Medicines Agency (EMA) now considers the Stride Velocity 95th centile (SV95C) as a new measure (known as a primary endpoint) to assess the effectiveness of trials of treatments for Duchenne muscular dystrophy (DMD). It can be used to assess the walking (ambulation) of children with DMD who are aged four and above.
Digital measure
The SV95C is a digital endpoint, which is a new way of assessing the impact of treatment on a person. It collects data about a person’s daily activity via a wearable device such as sensors. It means that they don’t have to be assessed in a clinic.
It is the first digital measure that has been qualified by a regulatory agency for use as a primary endpoint in pivotal studies. These are studies that are used by regulatory agencies to decide whether or not to approve a treatment.
Replacing the Six Minute Walking Test
It can now be used to replace the Six Minute Walking Test (also called the Six Minute Walking Distance) as a measure of the effectiveness of a treatment.
This is an important development as the Six Minute Walking Test has its limitations as a measurement as it relies heavily on how a child is feeling on that specific day of the appointment. It can therefore be an unreliable indicator of how a child may or may not be responding to a treatment overall.
Importance for DMD drug trials
This new measure will help the development and measurement of drug trials of DMD treatments. It could potentially shorten trial durations, and decrease enrolment requirements for them which would allow more children with DMD to take part.
DMD community achievement
This achievement is the result of a fourteen-year collaboration between many people across the DMD community which we were a part of.
We have promoted the use of wearable devices which children can wear on their wrist or ankle to use as a digital endpoint. This is because the continuous data collected from wearable devices at home could detect more sensitive and subtle changes and provide more reliable results about the impact or not of a new medicine.
In 2016, we awarded funding to support the EMA qualification procedure of the technology ActiMyo. This is a device which has been developed to evaluate the physical condition of patients with movement disorders. ActiMyo is the result of a close collaboration between the Institute of Myology and SYSNAV.
We are delighted that our support and the work of the DMD community has led to this new primary endpoint for DMD.