May 20th, 2021

// Vertex monopoly of the cystic fibrosis space signaled by another label expansion, says GlobalData

Vertex monopoly of the cystic fibrosis space signaled by another label expansion, says GlobalData

Vertex, the current owner of the entire cystic fibrosis transmembrane conductance regulator (CFTR) modulators’ market share, is expected to remain the market leader over the next decade, says GlobalData, a leading data and analytics company.

Mandana Emamzadeh, PhD, Healthcare Analyst at GlobalData, comments: “Many pipeline products have emerged in the early stage of development and Vertex will need to protect its position as market leader in the future. However, the company’s current strategy of usurping nearly all market share prior to launch of any competitors will be a viable strategy to remain the dominant player for the foreseeable future.”

In late April, the European Commission (EC) approved a label expansion for Vertex Pharmaceuticals' Kaftrio for the treatment of cystic fibrosis (CF) in all patients aged 12 years and older who are heterozygous for the F508del CFTR mutation (F), regardless of the other mutation type - either gating (G) or residual function (RF) mutations. 

Emamzadeh continued: “Key opinion leaders (KOLs) interviewed by GlobalData asserted that Kaftrio is the most effective CFTR modulator, and the drug is recommended to nearly all patients as soon as they are eligible. KOLs further anticipated that the eligibility of Kaftrio will expand to more mutations across all ages gradually.” 

While Kaftrio is taking over the CF market, a reasonable concern among developers is whether there is room for more CFTR modulators. 

Emamzadeh added: “KOLs believe that the CF community supports more options for CFTR modulators in case patients develop intolerances, allergies or adverse events. In addition, further competition can drive down the drug price and therefore the opportunity remains for developers.

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

The current CFTR modulators in early stage development include Phase I/II trial for Translate Bio’s MRT-5005, and Phase II trials for 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). 



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