UK Watchdog Backs Alexion Pharmaceuticals Inc.'s "Very Expensive" Drug Soliris

September 4, 2014

By Krystle Vermes, BioSpace.com Breaking News Staff

Alexion Pharmaceuticals Inc. announced today that the National Institute for Health and Clinical Excellence’s Highly Specialized Technologies Evaluation Committee has reaffirmed the significant value of eculizumab for the treatment of atypical hemolytic uremic syndrome.

Also known as Soliris, eculizumab has been deemed one of the only effective treatments for atypical hemolytic uremic syndrome, which can be life-threatening. There is a lack of other effective therapies on the market. As a result, NICE has issued a recommendation that Soliris be nationally commissioned in England for patients living with the disease.

The NICE EC confirmed the 2013 Advisory Group for National Specialized Services’ positive assessment of Soliris, as well as the NICE assessment from earlier this year, in its second Evaluation Consultation Document. The document was released today.

“We are pleased that NICE has once again confirmed that patients with aHUS are at constant risk of sudden, progressive and life-threatening damage to vital organs including the kidney and other organs, and that eculizumab is a significant breakthrough for patients with this devastating disorder,” said Leonard Bell, M.D., Chief Executive Officer of Alexion.”

At the moment, patients who have atypical hemolytic uremic syndrome are able to receive eculizumab through an interim policy that was commissioned last year. NICE has confirmed that the policy will remain in place until its appraisal is finalized. Alexion expects this to happen after a public EC meeting is held in early October.

“Alexion supports the use of eculizumab consistent with the EMA-approved label, which specifically directs that treatment is recommended to continue for the patient’s lifetime, unless the discontinuation of eculizumab is clinically indicated,” Bell continued. “We believe that it is important that NICE work within its remit and that decisions regarding continuation of eculizumab should be made by the treating physician based on best clinical judgment. We will provide specific comments to NICE to address its conditions and look forward to working to ensure equity such that patients with aHUS in England, like patients with PNH in England, have equal and sustained access to this life-transforming therapy.”

Atypical hemolytic uremic syndrome is a rare genetic disease that can be life-threatening if not addressed. The disease is caused by an uncontrolled activation of the complement system, which is a component of the immune system. Abdominal pain, vomiting and nausea are all symptoms of the condition.