Vertex gaining momentum with Kalydeco

Eyebrows were raised when Vertex decided to exit its hepatitis C franchise last year and concentrate its efforts on cystic fibrosis (CF), but with use of its lead product Kalydeco expanding quickly, the strategy seems to be paying off.

Vertex was facing a boom-and-bust scenario when its massively successful hepatitis C virus therapy Incivek (talepravir) started to be superseded by newer drugs, causing its sales to plummet and forcing the company to cut its workforce last October.

Sales of Incivek declined throughout 2013 and were just $4 million in the first quarter of this year, down from $205 million a year earlier and some way off its $1.5 billion-plus peak.

Many companies would have gone under in that situation but thankfully for Vertex Kalydeco (ivacaftor) has taken up the slack, with $100 million in sales in the first quarter and – it seems – plenty more to come.

Kalydeco was initially approved in 2012 to treat patients aged over six with a single mutation of the cystic fibrosis transmembrane regulator (CFTR) gene – known as G551D – which affects around 2,000 CF patients worldwide.

Sales have ramped up quickly – rising more than 60 per cent in the first quarter of 2014 on the back of reimbursement approvals in Europe – and Vertex now believes most eligible G551D patients are on the drug in countries where it is approved for sale. There are a couple of notable exceptions – namely Australia and Canada where reimbursement negotiations are ongoing.

The upside for Kalydeco is coming from the expansion of the drug’s use to include patients with other CFTR mutations.

Earlier this year, the US FDA approved Kalydeco for eight additional mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D), adding another 400-or-so patients to the drug’s target population. The EU’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion for these additional mutations in June.

Vertex has just filed for approval of Kalydeco for CF patients aged 18 and older who have the R117H mutation, the most common residual function mutation, which could add another 300 patients to the tally. EU filing for R117H is due in the third quarter.

Moreover, two pivotal trials have shown that combining Kalydeco with a second Vertex drug called lumacaftor (VX-809) – is effective in treating patients aged 12 or more with two copies of the F508del mutation, a particularly challenging defect to treat which accounts for around 60 per cent of all CF cases.

In North America, Europe and Australia alone there are more than 22,000 people aged 12 and older who have two copies of the F508del mutation.

Vertex expects to file for approval of the combination in the US and Europe before the end of the year, and analysts at ISI Group now predict that – notwithstanding any potential wobbles with regard to reimbursement or pricing pressure – Kalydeco and lumacaftor could rack up $3 billion a year in peak sales.

That represents not only a replacement for Incivek in terms of scale, but a replacement with the potential to bring in revenues over the long-term.

Vertex’ outgoing R&D chief Peter Mueller – who has just announced his retirement – must feel he is leaving the company in fine shape.


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