Sanofi is 1-1 in the U.K., with early access for dupilumab but a NICE 'no' for Cerdelga

London
England's NICE turned down a Sanofi rare disease med on Tuesday.

It's a good news, bad news week for Sanofi in England, where cost watchdogs turned away costly rare disease med Cerdelga. Despite that hit, the French drugmaker scored a win on its blockbuster hopeful dupilumab.

In a preliminary decision, experts at England’s NICE recommended against (PDF) use of the med in patients with type 1 Gaucher disease, citing “concerns about the true value for money” provided by the oral option.

On the other side of the coin, Sanofi announced on Tuesday that eligible atopic dermatitis patients in the U.K. will be able to receive dupilumab through the Early Access to Medicines Scheme even before the candidate secures a marketing approval.

In its Cerdelga draft guidance, NICE said Sanofi's oral therapy could provide “valuable quality-of-life benefits,” but the institute had to consider the “substantial additional costs” associated with the med, according its evaluation documents. As NICE noted, Gaucher disease is usually treated through intravenous enzyme replacement therapy, a “time consuming and burdensome” process.

Using Cerdelga would cost £84,559 in the first year of treatment based on list prices, according to NICE, and cost £571,487 by year 5. Sanofi offered a discount, but it wasn’t enough to win a coverage recommendation.

Sanofi said the announcement is a “disappointment,” but it will continue to advocate for its med.

“We are taking immediate action to better understand NICE’s position and the issues that led to this provisional recommendation.” Genzyme U.K. & Ireland general manager Peter Kuiper said in a statement.

In a Facebook post on the decision, patient group The Gauchers Association said it “will be working with clinical experts to challenge this decision.”

If all goes as planned, though, dupilumab's top-line contributions will be more than enough to offset potential impacts from the negative decision. EP Vantage predicted the med—to be marketed as Dupixent—will rake in $4.1 billion by 2022.