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Vertex scores FDA approval for CF triple therapy Trikafta

Decision comes only a few months after therapy was filed


Vertex has scored an earlier than expected FDA approval of its cystic fibrosis triple therapy Trikafta, and has priced it at $311,000 for a year of treatment. 

The combination regimen of VX-445 (elexacaftor), tezacaftor and ivacaftor has been approved for CF patients aged 12 and older with one F508del mutation and one minimal function mutation and in people with two F508del mutations. The approval comes only a few months after Vertex filed the triple therapy with the FDA.

This means that the therapy could help up to 27,000 CF patients in the US – around 90% of sufferers – compared to Vertex’s existing treatments – Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor) and Symkevi/Symdeko (tezacaftor/ivacaftor) – which can treat around half of patients.

“Today marks a milestone for CF patients, their families and Vertex. After a 20 year journey together, we have received FDA approval of Trikafta: a single breakthrough medicine with the potential to treat up to 90% of all people with CF in the future. For approximately 6,000 people with CF in the US, Trikafta is the first medicine that can treat the underlying cause of their disease,” said Jeff Leiden (pictured below), president and CEO of Vertex.

Jeff Leiden

Jeff Leiden, president and CEO of Vertex

Prior to this approval, there had been expectations that Vertex could set a lower price for the triple therapy thanks to this expanded patient population, but the pharma giant likely set the price higher as it attempts to recoup its years of R&D spending and investor backing.

The list price of $311,000 is more than Leerink analyst Geoffrey Porges' prediction that the triple therapy could be priced similarly to Symdeko, which costs $233,000 for a year’s supply in the US.

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.

Vertex has also submitted the triple therapy to the European Medicines Agency, with a decision expected in 2020.

It is also evaluating Trikafta in an ongoing phase 3 study of people aged six to 11 with F/MF and F/F mutations, while it has also said it is committed to evaluating the therapy in children under the age of six as part of planned future studies.

The approval is likely to increase pressure on England’s NHS and cost-effectiveness watchdog NICE’s to reach an agreement on Vertex’s previous CF drug Orkambi, following a long row over pricing. Trikafta is priced similarly to Kalydeco – Vertex and NHS England (NHSE) reached a pricing deal for this first CF drug in 2013.

However, since then NHSE’s specialised commissioning head John Stewart has said that it overpaid for Kalydeco. With the subsequent rows over Vertex’s new medicines, it looks likely that these will also extend to the triple therapy.

Article by
Lucy Parsons

22nd October 2019

From: Regulatory



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