Earlier this year, ProQR announced results from its phase 3 trial of sepofarsen and explained the company’s intention to consult with medicines regulators in the USA and Europe to clarify what might need to be done to get the treatment licensed for general use; more details are available in our April news story: Research update - ProQR.
The European Medicines Agency (EMA) has now recommended an additional clinical trial be conducted for sepofarsen for it to have the best chance of receiving marketing authorisation. As a result of this, ProQR has decided that it is no longer in a position to be able to take sepofarsen and ulteversen any further through clinical development, and that a partner taking over the two programs provides the best opportunity to bring these therapies to those living with inherited sight loss.
According to ProQR, in order to preserve operating capital, the company has had to suspend sepofarsen and ultevursen trial activity while it seeks a suitable partner. However, they have already manufactured a reasonable supply of both treatments and hope this will be enough to allow participants in the UK to continue receiving treatment, if they wish to do so, until the trials can be re-started by a future partner organization. Trial site clinicians have been in direct contact with participants to explain this latest development and next steps.
ProQR welcomes queries from the inherited sight loss community. If you would like any more information please contact them directly via email@example.com.
The Retina UK Helpline can provide information and support and is answered by volunteers living with inherited sight loss. The Helpline can be contacted on 0300 111 4000 (9.30am to 9.30pm on weekdays) or emailed via Helpline@RetinaUK.org.uk.