🧪 Why New Antifungal Trials Start with Invasive Aspergillosis

When you hear about promising new antifungal medicines such as Olorofim or Fosmanogepix, you may wonder why the first studies always seem to involve people with invasive aspergillosis — not those with chronic pulmonary aspergillosis (CPA) or allergic bronchopulmonary aspergillosis (ABPA).

It might seem unfair, especially when chronic forms of aspergillosis are so common and long-lasting.
But there are good reasons why research has to begin with invasive disease.

Here’s how it works — and why it’s still good news for everyone living with aspergillosis.

⚠️ 1. Invasive Aspergillosis Is the Most Dangerous Form

Invasive aspergillosis happens when Aspergillus spreads deep into the lungs or bloodstream, usually in people with a very weak immune system — for example, after chemotherapy, transplant, or high-dose steroid use.

Without prompt treatment, it can be fatal within days or weeks.
Because it is so serious, regulators such as the MHRA (UK), EMA (Europe) and FDA (USA) allow new drugs for invasive infections to be tested and reviewed much faster than they would for less urgent diseases.

This approach means that if a new antifungal proves helpful and safe, it can reach patients in greatest need more quickly — often saving lives while also building the data needed for later studies in other conditions.

📈 2. It’s Easier to Measure Whether the Drug Works

For invasive disease, the goal is very clear:

The infection either clears up, or it doesn’t.

That makes the results of a study straightforward to interpret.

With chronic or allergic aspergillosis, improvement takes much longer to measure:

• Scans may take months to show change,

• Symptoms can fluctuate naturally, and

• Other lung problems (like COPD or bronchiectasis) can confuse the results.

So trials in chronic disease need larger patient numbers and longer follow-up, which are expensive and take years. Starting with invasive aspergillosis lets researchers get the essential safety and efficacy answers first.

🧾 3. The Regulatory Framework Focuses on Invasive Disease

Drug-approval rules for antifungals were originally designed for the most life-threatening infections.
Official guidance documents — from the EMA, FDA and others — describe exactly how to test new drugs for invasive fungal infections, but there are no formal international standards yet for chronic or allergic aspergillosis.

That means developers start where the rules are clear — and then adapt once regulators, researchers, and clinicians agree on what a “successful outcome” looks like for chronic disease.

⚖️ 4. Safety and Ethics Come First

When a new antifungal is in early testing, doctors don’t yet know all its side-effects or how it behaves during long-term use.
For ethical reasons, it’s safer to begin in patients with very few other treatment options, where the potential benefit outweighs the risk.

As safety data builds up — including how the medicine interacts with other drugs — it becomes safer to test in people with more stable chronic conditions such as CPA.

🩺 5. Once Proven Safe, Use Can Expand

Once a drug like Olorofim or Fosmanogepix:

• works well in invasive aspergillosis,

• has solid safety data, and

• earns its first licence,

the manufacturer and research partners (such as the National Aspergillosis Centre) can propose new studies in CPA or other forms of aspergillosis.

By then, regulators already know the drug’s risk profile, dosing, and monitoring needs — so further approvals for chronic disease can move faster.

🧩 In Summary

🌱 Looking Ahead

Starting with invasive aspergillosis is a gateway, not a dead-end.
Every study adds vital knowledge about how these new antifungals work, how safe they are, and which patients might benefit most.

Once enough evidence exists, clinical trials can — and almost certainly will — expand to include chronic pulmonary aspergillosis (CPA) and possibly even allergic forms of the disease.

So while the research focus may begin with the most critical cases, the progress made there ultimately helps everyone living with aspergillosis.

by GAtherton

🧬 From Scottish Discovery to American Pharmacy Shelf: The Story of Brensocatib

Sometimes a medicine begins life in one country but reaches patients first in another. The new bronchiectasis drug brensocatib is a perfect example — discovered in Scotland, yet first approved for use in the United States.
Here’s how that happens, and what it tells us about how new treatments make their way to patients.

1️⃣ Discovery in Dundee

At the University of Dundee, scientists in the Drug Discovery Unit (DDU) were studying how certain white blood cells called neutrophils can cause long-term lung damage.
They identified an enzyme, DPP1 (dipeptidyl peptidase I), that activates destructive substances inside these cells.
Blocking DPP1 could calm inflammation without wiping out the body’s defences.
Their research produced a promising new compound — later named brensocatib — which safely switched off this process in lab studies.

2️⃣ Partnering to Go Global

Turning an early discovery into a medicine is an enormous task.
It costs hundreds of millions of pounds and can take 10–15 years.
The Dundee team partnered with Insmed, a biotechnology company based in New Jersey, USA, which had the funding and international trial experience to move brensocatib into large clinical studies.

3️⃣ Worldwide Trials

Insmed led major trials involving hundreds of people with non-cystic fibrosis bronchiectasis in hospitals across North America, Europe, and Asia.
Results showed that brensocatib reduced flare-ups and improved quality of life.
Because Insmed’s main offices and regulatory team are in the U.S., they submitted their results first to the U.S. Food and Drug Administration (FDA).

4️⃣ U.S. Approval

In 2025, the FDA approved brensocatib — the first drug of its kind to treat bronchiectasis.
American patients can now access it while other countries complete their reviews.

5️⃣ What Happens Next in the UK

In the UK, every new medicine goes through two steps:

• The Medicines and Healthcare products Regulatory Agency (MHRA) checks that it is safe and effective.

• Then NICE (the National Institute for Health and Care Excellence) reviews how well it works for its cost and decides whether the NHS should fund it.

NICE is expected to make its decision on brensocatib in July 2026.
Even if approved, it may first be offered to those with the most severe or frequent flare-ups while more real-world data are gathered.

💷 What Dundee Gained from Its Discovery

Although Dundee handed over development to a U.S. company, the university continues to benefit in several ways:

• Financial return: Dundee receives upfront payments, milestone fees for each stage of progress, and royalties on global sales.
  These funds support new drug discovery projects, student training, and research facilities.

• Scientific impact: Brensocatib’s success highlights the strength of the Drug Discovery Unit’s model, showing that UK universities can produce world-class medicines.

• Future partnerships: Dundee’s achievement attracts new collaborations and investment, ensuring that more early discoveries have a route to reach patients.

So while the drug is made and sold by Insmed, Dundee’s scientists — and their reinvested funding — continue to play a role in future breakthroughs.

🏭 Manufacturing: Turning Discovery into a Real Medicine

Once a new drug is approved, it still has to be produced safely, at scale, and consistently.
This is often a completely separate operation from the research or licensing stage.

For brensocatib, the chemical process that makes the active ingredient was developed by Dundee and Insmed scientists early on, but large-scale manufacturing is now carried out by specialist pharmaceutical plants under strict international standards known as Good Manufacturing Practice (GMP).

Because brensocatib is a small-molecule oral drug (a tablet, not an injection), it’s made in high-tech chemical manufacturing facilities, not hospitals or biologics plants.
These sites are often in Europe, the U.S., or Asia, depending on where the supply chains, raw materials, and quality-control systems are strongest.

Manufacturing is expensive — it must ensure every tablet is identical in purity, strength, and safety — but it’s also where economies of scale help keep the cost manageable once global production ramps up.

For the NHS and NICE, manufacturing details matter too, because:

• They affect cost-effectiveness (how much the NHS will pay per course of treatment).

• They influence availability — whether the company can supply enough medicine to meet demand once approved in the UK.

So, while the discovery began in Dundee and the approval started in the U.S., manufacturing is the bridge that makes it real — transforming a scientific idea into a medicine that can be prescribed to patients worldwide.

🌍 Why This Matters

This journey shows how scientific discovery is global.
A breakthrough can start in a Scottish laboratory, be developed with American funding, tested around the world, manufactured across several continents, and eventually come back to help patients in the UK.
It’s a reminder that international collaboration — between researchers, funders, manufacturers, and regulators — is what turns good science into real treatments.

by GAtherton