CSL Vifor plans UK launch after NICE backs rare disease drug Tavneos

UK cost-effectiveness organisation NICE has recommended routine NHS use of a recently-approved therapy for two forms of ANCA-associated vasculitis (AAV) – oral complement C5 inhibitor Tavneos – which will be launched in the next few weeks.

CSL Vifor – which licenses Tavneos (avacopan) from US biotech ChemoCentryx – announced the decision on behalf Vifor Fresenius Medical Care Renal Pharma, its joint venture with Fresenius, which will market the drug in the UK.

NICE has backed the use of Tavneos in combination with rituximab for adults with severe, active granulomatosis polyangiitis (GPA) or microscopic polyangiitis (MPA), the two main forms of AAV, which is a rare disease caused by inappropriate activation of the complement pathway that leads to inflammation in organs including the kidneys.

In a final appraisal document, NICE said it based its decision on data showing that Tavneos plus rituximab is more effective than standard treatment – cyclophosphamide or rituximab, followed by maintenance treatment with azathioprine or rituximab – at stopping the conditions getting worse.

It also reduces the need for corticosteroids, which are also used throughout treatment of GPA and MPA to control symptoms.

Tavneos was authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) in May, shortly after the European Commission cleared it in the EU, so on launch, it will become an option for patients in England and Wales, as well as Northern Ireland, which remains within the EU regulatory system.

In Scotland, patients will have to wait for a decision by the Scottish Medicines Consortium (SMC), NICE's counterpart north of the border. CSL Vifor has agreed to supply the drug at a confidential discount to its list price of £5,547.95 per pack of 180 x 10mg capsules.

The positive opinion from NICE has been welcomed by patient organisation Vasculitis UK, which said that AAV "can be rapidly fatal if not appropriately and promptly treated." The charity's vice chair, John Mills, said that the decision "means that eligible patients and clinicians have another choice of treatment for this debilitating condition."

Tavneos was approved in the US last October, prompting Amgen to table a $3.7 billion bid to buy out the biotech earlier this month.

Analysts have previously suggested that Tavneos could become a blockbuster product with peak sales of $1 billion or more if it gets approved in follow-up indications CE glomerulopathy – a rare kidney disease – and chronic inflammatory skin disorder hidradenitis suppurativa.