As the FDA mulls over SpringWorks’ experimental neurofibromatosis type 1 plexiform neurofibromas (NF1-PN) drug mirdametinib during its rolling submission, the biotech isn't wasting time in getting the awareness message about this rare disease out into the world.
Launched amid NF Awareness Month this May, its new campaign “Coping isn’t Care” is aimed at boosting knowledge of NF1-PN.
This rare disorder is a genetic condition that causes tumors to grow on nerves throughout the body and can manifest differently from patient to patient, making diagnosis difficult.
Sometimes it can be highly visual, presenting as dozens, and sometimes hundreds, of lumps on a patient’s skin. Other times it can be internal, making it harder to discern its impact on each patient.
Around 100,000 patients are estimated to be living with NF1 in the U.S. and an estimated 30-50% of those patients have plexiform neurofibromas.
NF1-PN can manifest in a variety of symptoms across numerous organ systems, including abnormal pigmentation, skeletal deformities, tumor growth and neurological complications, such as cognitive impairment. Patients with NF1 have an eight to 15-year mean reduction in their life expectancy compared to the general population.
The campaign, which has seen the launch of two new websites aimed at both patients and doctors, focuses on a condition that “can be especially challenging for adults,” the company said in a statement to Fierce Pharma Marketing.
This is because as children, the coordination of NF1-PN care is likely managed by their caregivers or within an NF clinic, but adult patients have fewer options for where they can receive specialized care.
“The Coping Isn’t Care campaign aims to empower young adult patients, who frequently drop out of care, to advocate for themselves and re-engage in ongoing care,” the company said.
The patient website includes personal stories and videos of people who have the condition. They talk about the disease and reinforce the message that the “adjustments you make [for the disease] are not an alternative to the care you need.” Similar messaging appears on the website for healthcare practitioners.
SpringWorks has kicked off a rolling submission for a potential approval of mirdametinib, its MEK inhibitor, in both child and adult patients with NF1-PN. The application is based on data from the phase 2b ReNeu trial, with data from that to be fully revealed at the American Society of Clinical Oncology conference, which begins May 31 in Chicago.
AstraZeneca’s Koselugo was approved in 2020 by the FDA for children with NF1-PN, making it the first specific drug to get the green light. SpringWorks hopes to gain a broader license to also treat adults.
The idea behind the campaign is to boost awareness in patients and docs not just about the disease, but the need for treatment, something that SpringWorks will hope will create a pathway to its drug, should it gain approval.