UK price watchdog after award winks at Alexion’s Ultomiris for second rare blood disease

By reducing its cost, Alexion obtained a second indication in the UK for its rare blood disorder drug Ultomiris.

A month after recommending Ultomiris to patients with paroxysmal nocturnal hemoglobinuria (PNH), the National Institutes for Health and Care Excellence (NICE) in England also approved its use for atypical hemolytic uremic syndrome (HUS).

The deal will make 160 HUS patients in the UK eligible to receive Ultomiris, NICE said. Last month’s recommendation opened up use of the drug to 270 people with PNH.

Since receiving FDA approval for PNH in 2018 and for HUS the following year, Ultomiris has been in rapid use around the world. It achieved blockbuster status last year, with sales reaching $ 1.08 billion, up from $ 339 million in 2019.

RELATED: Alexion’s Dear Ultomiris Gains Support From English Medicine Cost Watchdogs With Confidential Discount

But there was just one problem with Britain – its price.

Administered every eight weeks, an annual regimen of Ultomiris costs around £ 300,000. But earlier this year, Alexion and NICE agreed to a confidential discount.

Prior to NICE recommendations, there was only one treatment option for the two life-threatening disorders in England, Ultomiris predecessor, Soliris. The advantage of Ultomiris is that its infusion treatments are needed every eight weeks instead of two.

“Living with a rare blood disease can be both physically and mentally difficult, especially if frequent treatment is required,” Meindert Boysen, director of the Center for Health Technology Evaluation at NICE, said in a statement. “We hope that increasing the time between doses with this new treatment will lead to a better quality of life.”

Besides the advantage of the dosage, in PNH, Ultomiris is at least as effective and has been shown to cause fewer episodes of ruptured hemolysis, a symptom of the disease, reducing the number of hospitalizations and the need for blood transfusions.

Ultomiris is also cheaper, even before the handover, than Soliris, which ranked # 5 on a list of the most expensive drugs in the United States in 2019.

RELATED: FDA Gives Alexion Quick Deal to Track Soliris Ultomiris

PNH is characterized by a breakdown of red blood cells, which can lead to anemia, kidney problems, and clots in the blood vessels. HUS also causes clots in blood vessels, which can lead to organ damage, thrombosis, heart failure, and brain damage.

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