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Vertex files cystic fibrosis triple therapy with FDA

A landmark for company - but pricing controversy awaits


Vertex has hit a major milestone with the filing of its triple combination therapy with the FDA, which promises to expand treatment to up to 90% of all cystic fibrosis patients worldwide.

If approved, the triple therapy could transform treatment of the disease – and Vertex’s fortunes with it.

The three-in-one pill looks odds-on to gain FDA approval, having been granted a breakthrough therapy designation in 2018, and is likely to be granted a priority review to speed up the decision-date, which is expected to come by early 2020. A filing in Europe is also expected to follow shortly.

However also certain will be controversy around the pricing of the new combination, especially in the UK, (which has one of the biggest CF populations in the world) where there is a row over access to an existing two-in-one combination, Orkambi.

The combination regimen of VX-445 (elexacaftor), tezacaftor and ivacaftor is for CF patients aged 12 and older with one F508del mutation and one minimal function mutation and in people with two F508del mutations. This means whereas Vertex's existing treatments treat around 40,000 patients worldwide, the new triplet could help up to 68,000 patients, around 90% of sufferers.

Vertex is holding back details of the phase 3 trial results, but has confirmed two studies showed the combination hit its primary endpoint, statistically significant improvements in lung function (percent predicted forced expiratory volume in one second; ppFEV1), and all key secondary endpoints, with no new safety or tolerability concerns.

Just how significant these gains are will be important to healthcare systems, and will influence Vertex’s price in major markets.

Being able to reach a much expanded patient cohort should allow the company to set a lower price compared to existing treatments - Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor) and Symkevi/Symdeko (tezacaftor/ivacaftor), but the triple therapy is at the same time seen as Vertex’s big chance to recoup its years of R&D spending and investor backing.

Leerink analyst Geoffrey Porges last year predicted that the triple therapy could be priced similarly to Symdeko, which costs $233,000 for a year’s treatment in the US. However, independent US cost watchdog ICER concluded earlier this year that Symdeko needed to be 30% cheaper than this to be cost effective.

Analysts at William Blair earlier this year forecast Vertex’s CF franchise would eventually reach peak sales of $10.2bn, representing around 80-85% of the market. This forecast was boosted when its main challenger, Proteostatis Therapeutics, reported disappointing results for its rival triple therapy programme.


Reshma Kewalramani

"We have relentlessly focused on the progression of VX‑445 (elexacaftor), tezacaftor and ivacaftor from discovery through clinical development and regulatory submission,” said Reshma Kewalramani, M.D., executive vice president and chief medical officer at Vertex.

“The submission of the NDA is a major step toward our goal of bringing this medicine to the largest remaining group of people with CF that still do not have an approved Vertex medicine, as well as toward providing significantly enhanced benefits to patients with two F508del mutations. We will continue working with the FDA as they review the NDA and look forward to the potential of this triple combination regimen becoming a new treatment option for people with CF.”

The company will report its second quarter 2019 financial results on Wednesday 31 July.

Article by
Andrew McConaghie

23rd July 2019

From: Regulatory



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