News

Vtesse Receives Rare Pediatric Disease Designation by the FDA for VTS-270

Vtesse announced early in 2016 that the FDA granted Breakthrough Therapy designation status for VTS-270, which is currently in a pivotal Phase 2b/3 clinical trial in NPC. Both the FDA and the European Medicines Agency (EMA) previously granted Orphan Drug status to VTS-270, and the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA) granted it a Promising Innovative Medicine (PIM) designation.

"We are incredibly grateful to receive Rare Pediatric Disease designation status from the FDA for VTS-270, in addition to the Breakthrough Therapy designation status, Orphan Drug designations, and the MHRA PIM designation. These designations are granted to drugs with high promise that may address areas of unmet medical need or for rare diseases. These designations will aid us as we seek to develop VTS-270 as an effective treatment to slow the progress of NPC or stabilize patients who suffer from the devastating impacts of NPC," said Ben Machielse, Drs., President and Chief Executive Officer of Vtesse. "We recognize the considerable collaboration and efforts to advance the clinical development and regulatory processes in the rare disease space, and, above all, we continue to thank all of the parents who have supported the development of VTS-270."

Vtesse October 2016 Newsletter: Update on the Clinical Trial of VTS-270: The NPC Study

Here is the October 2016 edition of the Vtesse Newsletter, which highlights the most recent updates of the clinical trial of VTS-270. We have made it available in a number of different languages for our global community, including; Arabic, Dutch, English, French, German, Italian, Mandarin, Spanish EU, Spanish Lat. Am, and Turkish.

Sanofi Genzyme: Pivotal Phase 2/3 Trial of Olipudase Alfa for Adult Patients with Acid Sphingomyelinase Deficiency to Begin in the UK

Oxford, UK – 3 November, 2016 – Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the first adult patient in the UK has enrolled and been dosed in a pivotal Phase 2/3 clinical trial named ASCEND for the investigational therapy olipudase alfa. Olipudase alfa is an enzyme replacement therapy being studied for the treatment of non-neurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B (NPD B). The first patient was dosed at the National Hospital for Neurology and Neurosurgery at the University College of London Hospitals (UCLH).

Vtesse: Update on the Vtesse Clinical Trial of VTS-270 (September Newsletter)

Update on the Vtesse Clinical Trial of VTS-270:

We continue to open up new sites and enroll patients. We currently have 34 patients who have been enrolled in the trial.

Two sites in Australia are now active and both have patients set to screen in November.
The three sites in Germany report that they all have patients who will be screening in November.
We are working to bring interested families in from countries that are outside of the clinical trial site countries so that they can be involved in the trial.

Rare Diseases in General Practice: Recognising the Zebras Among the Horses

‘When you hear hoofbeats, don’t expect to see a Zebra.'

The above adage is especially useful in primary care as many of the conditions we see are common. But it is not so useful when we consider our role in both diagnosing and supporting patients with rare diseases. We must be ready to think again and look out for the horse with stripes.