What is expanded access? In advance of an official approval of a treatment for certain serious conditions, the FDA sometimes allows early use of the treatment in limited circumstances while it moves through the clinical trial process. PaxMedica is hopeful that PAX-101 will qualify for this designation, which would allow for the treatment to be introduced to a very small number of patients.
Should expanded access be granted, patients will need to apply and meet the eligibility requirements determined by PaxMedica. We expect that interest in the treatment may be greater than the limited supply through this program, and some of the supply will be reserved for clinical studies. VoxNova is committed to working closely with patients and families to set expectations and guide them through the process when the time comes.
Following is a quick look at what you can do now.
It’s important to start this journey with your child’s physician(s). First, ensure that your child has a formal diagnosis of ASD. If your physician is not familiar with PaxMedica and PAX-101 (intravenous suramin), we encourage you to direct them to PaxMedica.com to learn more.
For your convenience, we’re also providing you with an information sheet you can email or present to your physician on your next visit.
Physicians must be authorized by VoxNova to prescribe PAX-101. As physicians begin to go through the authorization process, we will provide a list on this page of those who are approved and open to contact for new patients.
If your physician would like to become an authorized PAX-101 prescriber to be considered for expanded access, they can apply directly on the Healthcare Providers page of this website by enrolling in the PAXREMS program.
Your authorized physician submits the PAX-101 prescription to Nufactor.
Nufactor assigns your child a client support specialist to uncomplicate the process, working with you, your physician and care team to explain the infusion procedure so you know what to expect and to guide you at every step.
Nufactor staff works with you to determine insurance coverage and explore eligibility for applicable Patient Assistance Programs (PAP) to lower out-of-pocket costs.
When you and your child arrive at your infusion center, the specially trained team will work with you to make the infusion experience as comfortable as possible. If needed, special care will be taken to calm children during the 30-minute infusion period.