How did BioMarin pick Brineura, this year's #FierceMadness drug-name champ?

One of BioMarin's CLN2 families. BioMarin's Brineura was approved to treat the rare and devasting brain disease last year. (BioMarin)(Source: BioMarin)

What’s in a winning drug name? BioMarin should know. For the second time, one of its products won our #FierceMadness drug name tournament—and we've only run two naming contests.

The winner is decided by voters who chose the best names from recent approvals in round-by-round match-ups leading to a final championship. In our 2015 naming contest, BioMarin's Vimizim won.

Brineura, this year’s BioMarin winner, beat AstraZeneca’s Imfinzi in the final match, thanks in large part to a united and dedicated effort from BioMarin employees who embraced, in their words, a "kumbaya" approach. While the final result was skewed by a few voting bots, Brineura had already trounced other names in round after round.

Comments from internal voters ran along these lines: “BRIghtens the future of patients with this severely debilitating and life threatening NEURological disease.” Meanwhile comments from non-BioMariners included “Looking at what each drug does, Brineura seems more on target—catchy too!”

In the end, what proved to be more important than BioMarin’s successful get-out-the-vote strategy was the name itself. We talked to Lauren Seilnacht, who led the BioMarin global marketing team for Brineura, and Debra Charlesworth, BioMarin’s vice president of corporate communications, about what went into selecting the Brineura name.

Everything about Brineura moved quickly, including the name approval, both agreed. Why the speed? Brineura treats CLN2 disease, a rapidly progressing and fatal brain disease that affects children. BioMarin garnered several FDA designations—an Orphan Drug Designation, a Breakthrough Therapy Designation and a Priority Review status—that helped move the drug more quickly than usual.

That meant the name needed to come quickly as well. BioMarin worked with the Brand Institute, which said it names more products than anyone else in the world. The Brand Institute worked on 84% of FDA-approved drug names and 77% of EMA-approved names in 2017, according to its website. The naming consultancy was also responsible for several other FierceMadness finalists including Tecentriq, Rebinyn and Symproic.

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In the case of Brineura, Brand Institute came back to BioMarin with hundreds of initial suggestions, but Brineura rose to the top from the beginning, Seilnacht said.

“We focused on what we wanted the name to stand for. We wanted it to be about brain and neurons, but we also wanted it to be about brightness and hope. This disease is a devasting disease and a really difficult diagnosis and we wanted it to have hopefulness in the name,” she said. “And we wanted it to sound like a pediatric drug that would sound accessible for families.” She added that another parameter BioMarin gave Brand Institute was a name that would work internationally and across many languages.

Brineura then made it through a gauntlet of requirements: The meaning had to make sense, couldn't be too close to another drug name in spoken or written language, and needed approval from various employees. So BioMarin applied to the FDA and the EMA for approval of the name.

Then they hit a snag. The EMA rejected the name, saying it sounded too much like another already approved drug. BioMarin had little time to start over, but moreover, really wanted the Brineura name. So for the first time in its history, the company went through a reversal; it appealed to the EMA to change its mind. Sielnacht and her team laid out all the reasons why Brineura was unique and the best name for the drug. In the end, the EMA agreed.

RELATED: BioMarin sees 'high end' pricing on Brineura ahead of FDA decision: analyst

Brineura was approved last April to treat CLN2 to slow the “loss of walking ability (ambulation) in symptomatic pediatric patients 3 years of age and older.” It is an enzyme administered directly to cerebrospinal fluid in the brain and is the first treatment for the disease—20 people are born with it in the U.S. every year. BioMarin estimates about 1,200 patients worldwide, though not all are diagnosed.

Patients have CLN2 from birth, but the average age for diagnosis is three. The rapid progression of the disease leads to the loss of the ability to walk and talk in young children in an ongoing downward spiral. Families often have more than one child with the disease because their families had grown before the genetic disorder was discovered in the firstborn.

One of the reasons for BioMarin employees' commitment to Brineura is that they know CLN2 families and the heartbreak of the disease. Two of the families with multiple CLN2 children attended an employee meeting last year to meet BioMariners and talk about their journey, participating in a panel discussion, Charlesworth said. Brineura's approval came too late for both families, who had already lost their older children to CLN2. And one of those families recently lost their second child.  

“We acknowledge the hope, but we also recognize the heartbreak,” Seilnacht said.