On 28 April, the European Commission (EC) approved the label expansion for Vertex Pharmaceuticals‘ Kaftrio (ivacaftor/tezacaftor/elexacaftor + ivacaftor) for the treatment of cystic fibrosis (CF) in all patients aged 12 years and older who are heterozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation (F), regardless of the other mutation type, either gating (G) or residual function (RF) mutations. Kaftrio was first approved on 21 August last year by the EC for CF patients aged 12 years and older with one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the CFTR gene.

Key opinion leaders (KOLs) interviewed by GlobalData asserted that Kaftrio is the most effective CFTR modulator and that the drug is recommended to nearly all patients once eligible. KOLs further anticipated that eligibility for Kaftrio will gradually expand to more mutations across all ages. While Kaftrio is taking over the CF market, a reasonable concern among developers may be whether there is room for more CFTR modulators. KOLs believe that the CF community supports more options for CFTR modulators in case patients develop intolerances or allergies or experience adverse events. In addition, further competition can drive down drug prices to the benefit of the consumers. The opportunity therefore remains for developers.

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There are, however, a couple of challenges that developers should expect to face when entering the CF market. Kaftrio has an established reputation and a firm position in the treatment paradigm. As a result, stable patients are unlikely to risk their condition getting worse by stopping a medication they are responsive to. As well as this, it is hard to recruit patients for new studies of this orphan disease when the majority of the population is already eligible for a marketed CFTR modulator.

CFTR modulators currently in early-stage development include Translate Bio’s MRT-5005 in Phase I/II development, and Eloxx Pharmaceuticals’ ELX-02, AbbVie’s ABBV-3067 alone and in combination with galicaftor (ABBV-2222), Yumanity Therapeutics’ dirocaftor (PTI-808) + nesolicaftor (PTI-428) + posenacaftor (PTI-801), Vertex Pharmaceuticals’ deutivacaftor (VX-561), and VX-121 + tezacaftor + deutivacaftor (VX-561). These are all in Phase II trials.

Vertex currently owns the entire CFTR modulators market share and is expected to remain the market leader over the next decade. But as pipeline products have emerged in early-stage development, Vertex will need to protect its position as market leader in the future. The company’s current strategy of usurping nearly all the market share prior to the launch of any competitors will be a viable strategy to remain the dominant player for the foreseeable future.

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