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FDA Approves Expanded Use Of Vertex Pharma's Cystic Fibrosis Drug In Younger Patients

fdaaprovedim sep19 09jun21 lt

The FDA has approved the expanded use of Vertex Pharmaceuticals Inc.'s (VRTX) blockbuster cystic fibrosis drug Trikafta in children of age 6 through 11 years old with certain mutations.

Trikafta was approved by the FDA for the first time in October 2019 to treat cystic fibrosis in patients aged 12 and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The drug generated sales of $3.86 billion in full year 2020, and according to a recent report by ResearchAndMarkets.com, Trikafta could achieve sales of about $5 billion by 2026.

Trikafta, which is also marketed in the EU as Kaftrio, is a CFTR Modulator and is the first triple combination therapy containing elexacaftor, ivacaftor, and tezacaftor. (CFTR means cystic fibrosis transmembrane conductance regulator).

The other three CFTR Modulators approved for cystic fibrosis include Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor) and Symdeko (tezacaftor/ivacaftor), all from Vertex' stable of drugs.

Royalty Pharma plc (RPRX) has the royalty rights to cystic fibrosis treatments developed by Vertex, which include Kalydeco, Orkambi, Symdeko/Symkevi and Trikafta/Kaftrio.

Royalty Pharma's royalty receipts from Vertex's cystic fibrosis franchise were $167 million in the first quarter of 2021, an increase of 68% compared to the same period of 2020.

VRTX closed Tuesday's trading at $211.13, down 0.94%. RPRX closed the day's trading at $46.44, down 0.04%.

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