This petition is for the implementation of a special access program for tovorafenib as monotherapy for pediatric, adolescent, and young adult patients in Canada who are battling recurrent, relapsed or progressive low-grade glioma (LGG) with a known RAF alteration. Given the urgent medical needs of these vulnerable patients and the limitations of existing therapies, we urge Ipsen Canada, DayOne Pharmaceuticals and Health Canada to facilitate access to this targeted therapy through a special access program.

The Burden of Disease

Pediatric, adolescent, and young adult patients with recurrent, relapsed or progressive LGG harboring RAF alterations face significant health challenges that impact not only their survival but also their quality of life, development, and future potential. LGG, while classified as a "low-grade" tumor, can be severely debilitating when progressive, leading to vision loss, motor impairment, seizures, cognitive decline, and chronic pain.

The Urgent Need for Access to Tovorafenib

Tovorafenib is an oral, selective RAF inhibitor with promising efficacy and a favorable safety profile for patients with RAF-altered tumors, including pediatric low-grade gliomas. Tovorafenib offers a targeted approach while effectively controlling tumor progression.

Preliminary clinical data suggest that tovorafenib can provide meaningful tumor control with improved tolerability, allowing patients the opportunity to continue their education, social interactions, and normal development with fewer interruptions from debilitating side effects. Given the urgent medical need and lack of optimal treatment alternatives for these patients, it is imperative that access be granted to this promising therapy through a special access program.

A Call for Action

As advocates for these young patients, we believe that every child, adolescent, and young adult deserves the best possible chance for a healthy future, free from the devastating side effects of the disease and harsh treatments. Special access programs exist to bridge the gap for patients who have exhausted standard treatment options and urgently require innovative therapies.

We urge Ipsen Canada, DayOne Pharmaceuticals and Health Canada to work collaboratively in ensuring that tovorafenib is made available to these patients through a special access program. These young individuals deserve hope, effective treatment, and the opportunity to thrive, and we implore you to take swift action to make this critical therapy accessible in Canada.

Sincerely,

Pam Jackson, a mother fighting for better treatment options for my 17 year-old daughter battling recurrent low-grade glioma