Patient landscape

What has been solved. What remains.

Published facts from the US Cystic Fibrosis Foundation Patient Registry 2024. This page is the leftover-disease picture. It is not a ranking and not a map of companies.

33,989

People in the Registry

US care-center headcount, not a market size

2,434

Cannot take a modulator

Ineligible by age or gene type. Many are too young. Use 1,226 for adolescents/adults with data.

623

Could take one, did not

Eligible in 2020, no modulator prescription 2022–24. Mixed reasons — not the same as the 2,434.

29.3%

Adults with CF-related diabetes

CFRD in adults. Do not apply this percent to 33,989.

12.1%

IV-treated lung flares

Pulmonary exacerbations. Was 35.8% in 2009.

23.3%

Pseudomonas in cultured patients

PA among people who had a culture. Was 52.0% in 2009.

80.2%

Still using pancreatic enzymes

PERT is a solved product class, not leftover CFTR.

19.2%

CFRD, complications table

Same diabetes, different slice (~32,492 people, not 33,989).

Solved vs leftover, by topic

Solved means a class that already works for most people. Remains is a countable leftover. Interpretation is what we think that means — still not a score.

CFTR function

N1 · Mutation-agnostic CFTR restoration · N2 · Eligible but not prescribed

Solved

After Alyftrek, CFF estimates ~92% of people with CF are eligible for at least one modulator. Among ages 12+, 87.5% were prescribed a modulator in 2024.

Still leftover

2,434 people were ineligible by age or genotype; 1,226 ineligible adolescents/adults had Registry data. 623 people aged 12+ in 2020 had no CFTRm prescription in 2022-2024.

What we think it means

Protein modulation is the solved core for most US patients. Remaining causal CFTR restoration is concentrated in no-protein / poorly responsive genotypes plus a smaller mixed access/tolerability group.

Survival and demography

N7 · Aging with CF

Solved

Predicted median survival rose from 38.0 years (2009) to 65.4 (2024). Adults are 61.6% of the Registry, versus 33.8% in 1994. People aged 60+ number 1,437.

Still leftover

226 deaths were still reported in 2024. Median age at death was 38.8 years. Adult anxiety 31.9% and depression 30.2%. Osteopenia 19.4% of adults.

What we think it means

CF is now an adult and increasingly older-adult disease in US care centers. That shift creates screening, metabolic, malignancy, and care-delivery questions; it does not by itself identify a biotech company.

Pulmonary exacerbations and lung function

N3 · Residual lung disease

Solved

IV-treated pulmonary exacerbations fell from 35.8% (2009) to 12.1% (2024). Mean FEV1 rose from 79.0% to 89.1% predicted among ages >7.

Still leftover

Exacerbations did not reach zero. Mean FEV1 among ages 40-49 was 72.8% predicted. Lung transplants fell from 249 (2019) to 61 (2024), not to zero. Dornase alfa remained at 80.6% of ages >=6.

What we think it means

Highly effective modulators reduced pulmonary crisis rates. Residual lung disease, airway clearance burden, and advanced disease still exist and may be a crowded symptomatic-pipeline space.

Chronic airway infection

N4 · Chronic infection

Solved

P. aeruginosa prevalence among cultured patients fell from 52.0% (2009) to 23.3% (2024). MRSA fell from 24.0% to 13.1%.

Still leftover

Nearly one in four cultured patients still had PA. NTM was 11.6% of those with a mycobacterial culture and 18.7% among ages 60+. Inhaled tobramycin remained 51.2% of PA-positive ages >=6.

What we think it means

Infection burden declined with modulators and possibly with fewer cultures. Persistent PA, NTM in older adults, and ongoing inhaled-antibiotic use keep infection on the unmet-need list.

CFRD and metabolism

N5 · CFRD / diabetes and metabolism

Solved

CFRD is recognized, screened, and usually treated; ~58% of people with diabetes in the Registry used CGM. Insulin remains guideline standard of care.

Still leftover

CFRD prevalence was 19.2% in the transplant-censored complications population and 29.3% among adults. 16.4% of people with CFRD had no treatment noted. No CF-specific disease-modifying metabolic approval is in the MVP therapy table.

What we think it means

High adult prevalence plus insulin-centric care is a burden fact. Whether that is a CF-specific drug opportunity is a Phase 4-7 question, not a 2024 Registry conclusion.

GI disease and CF liver disease

N6 · GI and CF liver disease

Solved

PERT products are approved and widely used. Tube feeding fell to 5.1% in 2024.

Still leftover

80.2% still used PERT. Cirrhosis 2.7% and non-cirrhotic liver disease 3.5% in the complications population. UDCA was prescribed in 10.1%; CFF recommends against routine UDCA to prevent advanced CFLD.

What we think it means

Pancreatic enzyme replacement is solved as a product class but not as a disease. Advanced CFLD is smaller and severe. Endpoints and addressable population need diligence later.

Treatment burden and care delivery

N8 · Treatment burden and care delivery

Solved

Visit volume and some inhaled-therapy intensity declined after 2019. Telehealth exists (mean 0.4 visits in 2024).

Still leftover

People still averaged 3.0 clinic visits. Hypertonic saline 62.6% and dornase 80.6% among ages >=6. 60.4% saw a pharmacist. The population is now majority adult.

What we think it means

Burden fell but did not disappear. This is the opening for services/digital theses, not a default therapeutic white space.

Clinical development activity

Not a patient leftover — this card hands off to Pipeline and Map

Solved

ClinicalTrials.gov contains a large historical interventional corpus tagged to CF (1,209 records in this snapshot, retrieved 2026-09-09).

Still leftover

A heuristic flagged likely non-CF bronchiectasis tagging noise. Recruiting industry titles in the raw extract include multiple NCFB programs. 2026 start counts are a partial year.

What we think it means

Do not rank competitive intensity from unfiltered condition search. Phase 4 must review CF versus NCFB versus other muco-obstructive studies before whitespace scoring.