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Vertex's two-drug cystic fibrosis treatment cleared in US

FDA approves Symdeko, which combines Kalydeco and tezacaftor

Vertex PharmaceuticalsVertex Pharma has won an FDA green light for a third product for cystic fibrosis, and its second combination therapy for the genetic lung disorder.

The new drug - called Symdeko - combines the active ingredient in Vertex’ first CF therapy Kalydeco (ivacaftor) - first approved in 2012 - with new molecular entity tezacaftor and is a key launch for the company as it underpins its future plans for three-drug therapies.

It will start shipping the product in the US later this week with an annual price of $292,000, a little higher than the list price for Orkambi. Vertex has also filed for approval in the EU with a verdict due before the end of the year.

The new product is approved to treat CF in people aged 12 years and older who have two copies of the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene or who have at least one mutation that is responsive to tezacaftor/ivacaftor. It is estimated that makes around 45% of all CF patients eligible for treatment with the new product.

According to Vertex, ivacaftor enhances the function of a protein called CFTR at the cell surface, helping to improve the transport of salt and water across the cell membrane which in turn clears mucus from the airways. Tezacaftor works in a different way, increasing the amount of mature CFTR that reaches the cell surface by targeting defects in the processing and trafficking of the CFTR protein.

Vertex already markets a two-drug combination called Orkambi which combines ivacaftor with lumacaftor, which brought in $1.3bn in sales last year to overtake Kalydeco’s $845m turnover.

The new approval is expected to help the company increase its coverage of the CF population, as clinical data (albeit without a head-to-head assessment) suggest the duo has greater efficacy and is more tolerable than Orkambi, which has respiratory side effects that affect around a quarter of patients and can lead to therapy drop-outs.

Symdeko has the potential to win new prescriptions as well as provide an option for patients who find it difficult to tolerate Orkambi, with analysts at Jefferies saying they expect sales of $100m-$200m this year “based on identified patients who came off Orkambi in the last two years”.

Vertex’ chief executive Jeff Leiden said during the company’s annual results meeting that it “remains focused on bringing forward the best triple combination regimen to patients as quickly as possible”, adding that it intends to start phase III programmes for two triples in the first half of this year.

The company currently has a series of three-drug combinations that have reached phase II testing, including two that add new-generation CFTR correctors (VX-440 and VX-659) to Symdeko and according to Leiden “have highly compelling profiles for late-stage development”. It is also conducting trials with a regimen that swaps ivacaftor for another compound, VX-561, which could make once-daily dosing an option.

Article by
Phil Taylor

14th February 2018

From: Research



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