Vanishing White Matter

GuanaRep

Repurposing guanabenz for Vanishing White Matter, taking an academic discovery through clinical development, pharmaceutical development and a registration pathway designed around patient access.

Amsterdam UMC Guanabenz Phase I/II results published
Academic origin
Academic-driven development From clinical signal
to an accessible treatment.
Amsterdam UMC
01
Opportunity + development

A blood-pressure drug.
A potential VWM treatment.

Academic research at Amsterdam UMC identified guanabenz, an existing antihypertensive medicine, as a potential treatment for Vanishing White Matter.

The programme has progressed through preclinical research and a Phase I/II study. The development challenge now is to convert that clinical evidence into the pharmaceutical and regulatory package required for registration.

Academic
science
Programme
structure
Development
Clinical
progression
Patient
access
05

GuanaRep

Vanishing White Matter

02
What Orfenix adds

Connecting the trial to a route towards approval.

03
Access + next step

Clinical evidence in hand.
Registration is the next route.

Access by design

The goal is not simply approval. It is access for every VWM patient.

GuanaRep is organised as a public-private partnership, bringing academia, patient organisations, funders, investors and pharmaceutical specialists together around a socially responsible route to market.

01 Public-private partnership
02 Fair-price development
03 Global patient access
NEXT ↗
EMA Protocol Assistance

Defining the remaining registration pathway.

With Phase I/II clinical data available and the extension study continuing, the programme is using EMA Protocol Assistance to identify the remaining evidence required for marketing authorisation.

Built with Amsterdam UMC Amsterdam Leukodystrophy Center Hersenstichting Stichting RARE-NL