NICE issues final positive recommendation for national commissioning of Soliris for patients with atypical hemolytic uremic syndrome (aHUS)
Alexion Pharmaceuticals, Inc. has announced that the National Institute for Health and Care Excellence (NICE) Highly Specialised Technologies Evaluation Committee (EC) has recommended that Soliris® (eculizumab) be commissioned for all patients in England suffering from atypical hemolytic uremic syndrome (aHUS), a severe and life-threatening ultra-rare disorder. With this final evaluation determination, NICE has recommended Soliris for use within the National Health Service (NHS) in England as the first and only treatment for patients with aHUS.
“Today’s decision is an important victory for patients with aHUS and physicians who now have assurance that they will have access to the life-transforming efficacy of Soliris. We are pleased that Soliris will be made available on the NHS for patients with aHUS and we commend NHS England for its previous decision to provide interim funding to patients for the extended period it took for NICE to make this final determination,” said Leonard Bell, M.D., Chairman and Chief Executive Officer of Alexion.
In the final evaluation determination published yesterday, the NICE EC again confirmed that Soliris represents an important treatment option of significant value to patients with aHUS. The Committee recommended the commissioning of Soliris, within its marketing authorization, for aHUS subject to the conditions provided in the evaluation consultation document released in September. Soliris was approved in November 2011 by the European Commission for the treatment of patients with aHUS. The Clinical Particulars section of the EU label states that “Soliris treatment is recommended to continue for the patient’s lifetime, unless the discontinuation of Soliris is clinically indicated,” as described in the Special warnings and precautions for use subsection. [i] As noted by NICE in its final evaluation determination, after stopping Soliris, severe TMA complications were reported including graft failure needing hemodialysis, renal insufficiency, end-stage renal failure and respiratory distress needing intubation.
“Patients with aHUS are at constant risk of sudden, progressive and life-threatening damage to vital organs including the kidney and other organs,” said Keith Woods, Vice President and General Manager of Alexion Pharma UK. “Alexion supports the use of Soliris consistent with the EMA-approved label and firmly believes that decisions regarding continuation of Soliris should be made by the treating physician based on best clinical judgment.”
NICE recommended that the following arrangements be in place as conditions for the funding of Soliris for patients with aHUS:
- Coordination of the use of eculizumab through an expert center;
- Monitoring systems to record the number of people with a diagnosis of atypical hemolytic uremic syndrome, the number of people who receive eculizumab, and the dose and duration of treatment for these people;
- A national protocol for starting and stopping eculizumab for clinical reasons;
- And a research programme with robust methods to evaluate when stopping treatment or dose adjustment might occur.
[i] Soliris eMPC. Available at http://www.medicines.org.uk/emc/medicine/19966. Last downloaded on 20 Oct 2014.
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[iii] Ariceta G, Besbas N, Johnson S, et al. Guideline for the investigation and initial therapy of diarrhea-negative hemolytic uremic syndrome. Pediatr Nephrol. 2009;24:687-96.
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[vi] Noris M, Caprioli J, Bresin E, et al. Relative role of genetic complement abnormalities in sporadic and familial aHUS and their impact on clinical phenotype. Clin J Am Soc Nephrol. 2010;5:1844-59
[vii] Bresin E, Daina E, Noris M, et al. Outcome of renal transplantation in patients with non-Shiga toxin-associated hemolytic uremic syndrome: prognostic significance of genetic background. Clin J Am Soc Nephrol. 2006;1:88-99.