We are thrilled to let you know that this year, thanks to PRF’s many generous donors, our ONEpossible Campaign raised over $120,000!
With your generosity, children and young adults with Progeria can access the heart-strengthening, life-extending, FDA-approved drug Zokinvy, no matter where they live.
While we’re thrilled with this accomplishment, Zokinvy is a treatment and not a cure, so our work continues. Transformational research in gene therapy and drug development are ongoing and will carry us toward more effective treatments and the cure.
That’s our PURPOSE.
That’s our PROMISE.
That’s what each ONE of us will make POSSIBLE – together.
In 2020, PRF checked off an important mission milestone: U.S. Food and Drug Administration (FDA) approval of lonafarnib (brand name Zokinvy®), the first-ever treatment for Progeria. Since then, and through tremendous effort and expense, PRF and partner Eiger BioPharmaceuticals have achieved global access to Zokinvy through three pathways:
Meghan, age 21, U.S.
1. Prescription in the U.S.
Now that Zokinvy is FDA-approved, Meghan can conveniently access the drug through prescription at her local pharmacy.
2. Managed Access Program (MAP) in international countries where available
MAP allows Enzo and those outside of the U.S. with Progeria to obtain Zokinvy through their local physicians.
Enzo, age 10, Australia
Michiel, age 23, Belgium
3. PRF-funded clinical trials, for those residing in non-U.S., non-MAP countries
Because Michiel’s home country does not offer MAP, PRF continues to fund a 1-drug trial so he and others in similar situations can access Zokinvy.
We’re working tirelessly to ensure that every child and young adult with Progeria has access to this life-extending drug. But Zokinvy is a treatment, not a cure.
With YOUR continued support, we won’t stop until we have even better treatments and the cure.
