Neurocrine launches CAH rare disease education campaign ahead of FDA submission

Neurocrine Biosciences’ newly-debuted campaign acknowledges the frustration that congenital adrenal hyperplasia (CAH) patients, families and endocrinologists often feel.

The “What the C@H?!” theme is a tongue-in-cheek play on the popular “WTF” phrase, reflecting the difficulty managing the condition’s symptoms and treatments, but encouraging people not to be resigned to it.

“It’s a condition of both excess and absence,” Neurocrine’s chief commercial officer Eric Benevich said, noting the careful balancing act in dealing with both the non-production of cortisol and its triggering of excess production of androgen hormones.

Current treatment for the hormone deficiency is typically hydrocortisone for children and a combination of hydrocortisone and prednisone or dexamethasone for adults. An estimated 20,000 to 25,000 people ranging in age from infants to older adults are living with CAH in the US.

Eric Benevich

Neurocrine’s research with patients and focus groups discovered “a universal feeling that people had of, ‘Wow, this is really challenging and frustrating at times to manage this condition and finding the right balance between the illness itself and the treatment,” Benevich said.

The education and support-focused work includes separate but similar “What the C@H?!” websites for patients and healthcare professionals. Neurocrine plans to use social media, targeted advertising, patient advocacy groups and endocrinology conferences to drive people to the sites.

On the site, patients, caregivers and others can also upload their own stories and experiences, which will be vetted and shared. As with other rare diseases, patients don’t always know anyone else living with CAH, so the hope is the campaign will help people to connect and talk about their experiences, Benevich said.

While the new campaign is educational and unbranded, Neurocrine nabbed a breakthrough designation for crinecerfont after publishing successful late-stage study results. It plans to submit a new drug application to the FDA in the first half of this year.

Analysts estimate peak sales of the drug could reach $1 billion.