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They were the 10% left out of life-changing treatments. Now these cystic fibrosis patients may finally get help

Key Takeaway: Vertex Pharmaceuticals has significantly improved cystic fibrosis treatments, benefiting many patients. However, about 10% of patients remain without effective options due to specific genetic mutations. These patients continue to rely on older medications while their peers experience life-changing improvements. Vertex aims to address this gap in treatment.
Price reaction · baseline $15.77 (2023-09-14 close) · hit pre-market · 5 other FDMT headline(s) in the window, move may be shared
day 0 close · peak
-1.3%

Market Sentiment Analysis

POSITIVE FACTORS

  • Vertex Pharmaceuticals has transformed cystic fibrosis treatment.
  • Patients can abandon time-consuming treatments and breathe easier.
  • New treatments may soon benefit the 10% of patients left out.

BiopharmaWatch Analysis

From our catalyst data and publicly available data · not financial advice
Best trade, last catalyst
+128%
120-day peak, hindsight
Typical move
11.2%
average across 9 past catalysts
Cash runway
~18 mo
Low dilution risk
Lead asset
4D-150 IVT
Phase 3 · Macular Neovascularization Secondary to Age-Related Macular Degeneration

Full Press Release Details

V ertex Pharmaceuticals revolutionized the treatment of cystic fibrosis. It also has unfinished business.
The Boston drugmaker’s medicines for the genetic disease — which damages the lungs and other organs — have transformed the lives of patients, helping them abandon other time-consuming treatments, shrug off what would have been dangerous respiratory infections, and just live breathing easier. The drugs, a number of so-called modulators approved starting in 2012, have been one of the great biotech success stories .
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But the medications don’t work for some 10% of patients , depending on which CF-causing mutations they inherit. These patients have watched as their peers’ lives have changed, while they remain stuck using old medications to manage their symptoms and to slow the worsening of their illnesses.
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About the Author Reprints

Andrew Joseph

Europe Correspondent
Andrew covers the biopharma industry, scientific research, and public health across the continent. You can reach Andrew confidentially on Signal at drewqjoseph.71.

Frequently Asked Questions

What percentage of cystic fibrosis patients are left out of treatments?

About 10% of cystic fibrosis patients do not benefit from current treatments.

How have Vertex's drugs changed patients' lives?

Vertex's drugs have allowed patients to abandon old treatments and breathe easier.

What is Vertex Pharmaceuticals' goal for cystic fibrosis patients?

Vertex aims to develop treatments for the 10% of patients who currently lack effective options.

Last updated: Sep 15, 2023