The UK's National Institute for Health and Care Excellence (NICE) has rejected Kanuma (sebelipase alfa) for treating lysosomal acid lipase deficiency (LAL-D), dealing another blow to Alexion Pharmaceuticals' ultra-rare disease portfolio. This decision comes just eight days after NICE's rejection of Strensiq, marking a challenging period for the company's rare disease treatments in the UK.
Unprecedented Pricing and Cost Concerns
Kanuma's list price of £491,992 per patient per year makes it the most expensive drug ever reviewed by NICE, surpassing even Alexion's previous record-holder, Soliris. Despite Alexion offering confidential discounts and proposing an annual per-patient cost cap, NICE determined the price remained too high relative to the demonstrated benefits.
Professor Carole Longson MBE, NICE health technology evaluation centre director, emphasized the complexity of the evaluation: "Even their best offer to date falls far short given the considerably uncertainties about its longer term benefits and its very high cost."
Clinical Impact and Patient Population
LAL-D affects approximately 25 people in England and can be particularly devastating for infants under six months old. Patient experts testified that Kanuma represented a potential lifeline for babies with this condition who currently have no other treatment options. However, NICE's committee concluded that the treatment benefits remained uncertain despite these compelling patient needs.
Industry Implications and Market Access Challenges
Alexion has strongly criticized NICE's decision-making process, describing it as "inconsistent" and "ineffective." A company spokesperson warned of potentially "devastating consequences for patients in England with LAL-D who may face significant suffering, multi-organ damage and potential premature death."
The company argues this rejection highlights how England and Wales are falling behind other countries in funding medicines for rare and ultra-rare diseases with limited treatment options.
Future Outlook and Policy Context
NICE and NHS England have proposed new evaluation criteria for highly specialized technologies, including a QALY threshold allowing automatic funding for very rare condition treatments up to £100,000 per QALY. Treatments exceeding this threshold would require review through NHS England's prioritization framework.
Some hope remains for a potential agreement, as demonstrated by the eventual approval of Alexion's Soliris for atypical haemolytic syndrome (aHUS) in 2014, though that drug's annual cost of £340,000 per patient was significantly lower than Kanuma's current price point.
