The 2017 North American Cystic Fibrosis Conference (NACFC) was held at the Indiana Convention Center in Indianapolis, Indiana from November 2–4.

The conference annually brings together scientists, clinicians, and caregivers worldwide and facilitates discussion about ways to improve the health and quality of life for cystic fibrosis (CF) patients. Sponsored by the Cystic Fibrosis Foundation (CFF), the 2017 NACFC featured a wide variety of session formats, including didactic symposiums, interactive workshop sessions, brown bag luncheons, and grand rounds. Each day of the conference was highlighted by a themed plenary session headed by leading experts in the CF community.

CF is an inherited, autosomal recessive disorder that causes persistent lung infections and impairs breathing ability over time, and affects more than 70,000 people worldwide. Characterised by a loss of function in the cystic fibrosis transmembrane conductance regulator (CFTR) protein, CF causes abnormally thick, sticky mucus production that can lead to airway obstruction and lung infections, and can eventually lead to pancreatic insufficiencies due to mucous blockage of the pancreatic duct. Currently there are six different classes of CF that categorise more than 2,000 different CFTR mutations. The F508del mutation is the most prevalent CF mutation, a Class II mutation that accounts for approximately two-thirds of CF cases worldwide.

The first speaker of the conference presented an overview of the progress made in developing therapies for CF patients during the past year. Dr Preston Campbell, president and CEO of the CFF, announced that last year’s median predicted survival age of CF patients was 41 years. This year, that number jumped to 47 years. Campbell emphasised the significant impact that CFTR modulators, proteins that address the basic defect of CF, have brought to the community.

Vertex Pharmaceuticals, an important partner of the CFF since 1998, and the sole pharmaceutical company with marketed CFTR modulating therapies, received many favourable clinical trial results and rulings during 2017. Its lead CFTR modulator, Kalydeco (ivacaftor), received multiple label expansion approvals from the FDA to accommodate 28 additional CF mutations. As a result, approximately 1,500 CF patients ages two and older who have certain residual function mutations now have a CFTR modulating option as a part of their treatment regimen.

In addition, according to Dr Campbell, the positive Phase III results regarding the efficacy and safety of Vertex’s ivacaftor and tezacaftor combination therapy are very promising. This combination therapy already appears to have a more favourable safety profile than Vertex’s marketed therapy, Orkambi (ivacaftor/lumacaftor). Vertex’s early-stage next-generation correctors also appear to work effectively in combination with ivacaftor and tezacaftor.

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According to key opinion leaders (KOLs) interviewed by GlobalData, CF patients will still need symptomatic treatments, as CFTR-modulating therapies may not fully restore CFTR function. Campbell indicated that novel antibiotics, mucolytics, and anti-inflammatory therapies will still need to be developed to address the various symptoms that accompany CF.

Dr Campbell ended his segment of the plenary by explaining the meaning of “Until it’s done,” the slogan of the CFF. The CFF will not stop until every CF patient has a CFTR modulator. In order to get to this stage, the CF community will need to address the need for modulators for nonsense mutations and develop mutation agnostic therapies that enable production of a normal CFTR protein. Once every patient has a CFTR modulator, Campbell made it clear that the job still isn’t done. The CFF’s mission is to cure CF, so that patients can one day say, “I used to have CF, but not anymore.” He was confident that the CF community will continue to develop these essential treatments, while also employing gene editing research to pursue and achieve this dream.

Related reports

GlobalData (2017). OpportunityAnalyzer: Cystic Fibrosis – Opportunity Analysis and Forecasts to 2025, March 2017, GDHC065POA

GlobalData (2017). Expert Insight: Considerable Opportunity Still Remains for the Development of New CFTR Modulators and Curative Therapies, February 2017, GDHC1229EI

GlobalData (2016). Expert Insight: Obstacles Remain for Vertex’s Cystic Fibrosis Drug Orkambi, Despite FDA Approval for Label Expansion, October 2016, GDHC1128EI

GlobalData (2016). NACFC 2016 Highlights – A Crucial Need for Symptomatic Treatments Still Exists for Cystic Fibrosis Patients, December 2016, GDHC1152EI