Can Enhertu treat colorectal cancer? Latest results, approval timelines, and more.

Last updated: 15 January 2024

Since its first FDA approval in 2019, Enhertu has gained a lot of attention in the world of oncology. In 2023, Enhertu continues its impressive trajectory with two additional FDA breakthrough designations. It now boasts a total of seven ¹.

One of Enhertu's latest breakthrough designations is for the treatment of HER2-positive metastatic colorectal cancer ². For all colorectal cancer patients, this is an important milestone. At the same time, it poses a lot of questions.

Here's all you need to know about Enhertu for colorectal cancer.

Does Enhertu's breakthrough designation equal FDA approval?

No. Breakthrough Therapy designation doesn't automatically mean FDA approval. However, it has benefits, such as intensive guidance and senior management involvement in the medicine development process. These advantages can help speed up the subsequent approval of a medicine ³.

What types of cancer is Enhertu approved for?

Enhertu is currently approved by the EMA in Europe for the treatment of:

HER2-positive metastatic breast cancer, which cannot be surgically removed;
HER2-low metastatic breast cancer, which cannot be removed by surgery;
HER2-positive advanced gastric cancer (stomach cancer) or gastro-oesophageal junction cancer ⁴.

In the USA, Enhertu is additionally approved for the treatment of:

HER2-mutant metastatic Non-Small Cell Lung Cancer ⁵.

How effective is Enhertu for colorectal cancer?

Since Enhertu is a targeted therapy, it's only applicable to cancers with a HER-2 expression. About 3-5% of all colorectal cancers fall under this category⁶.

Clinical trial results

In June 2023, the final results of the DESTINY-CRC01 trial were published. This phase 2 trial focused on assessing the efficacy and safety of Enhertu in patients with HER2-expressing metastatic colorectal cancer. The patients in the trial had tumors that progressed after at least 2 prior rounds of treatment ⁷.

The key outcomes of this trial for colorectal cancer patients were:

45.3% of patients treated with Enhertu experienced partial shrinkage to their tumors;
The response to treatment was typically seen about 2 months after treatment start;
The median duration of response was 7 months;
The median progression-free survival was 6.9 months;
The median overall survival was 15.5 months. This result exceeds significantly the current standard of care. For instance, regorafenib and trifluridine/tipiracil are common third-line therapies. These treatments have an overall survival of 6.4 months and 7.1 months, respectively ⁷.

Another trial, called DESTINY-CTC02, is studying Enhertu for colorectal cancer patients. The trial is still ongoing, but its preliminary results are in line with the outcomes of the DESTINY-CTC01 trial ¹⁰.

How effective is Enhertu for colorectal cancer?

How safe is Enhertu for colorectal cancer?

In terms of safety, these are the main results from the DESTINY-CRC01 trial:

Each patient had a side effect from the treatment. The most common side effects were stomach and blood problems.
Serious adverse effects occurred in 37.7% of patients. The most common serious side effects were low neutrophil counts (22.1%) and anemia (14%) ⁷.
A side effect called interstitial lung disease (ILD) was associated with discontinuation of the drug and was observed in 7% of patients.
3 drug-related deaths were reported, all of which were associated with interstitial lung disease.

What does this mean?

Enhertu's safety results cannot be directly compared between different patient groups. However, to help you put things in context, here is a summary of Enhertu's safety results across multiple tumor types. Including tumors for which Enhertu is approved:

ILD occurred in 12% of trial patients, with fatal outcome in 1.5% of cases;
Decreased neutrophil counts occurred in 34.6% of trial patients;
Anaemia occurred in 34-43% of trial patients (depending on medication dosage) ⁸.

Considering this, the safety profile of Enhertu for colorectal cancer is similar to its profile for other approved cancers.

Enhertu

When will Enhertu be approved for colorectal cancer?

The approval of Enhertu for HER2-positive colorectal cancer has no clear timeline.

The DESTINY-CRC02 trial is ongoing, with final results expected in July 2024 ⁹. If results are positive, the FDA might approve Enhertu for colorectal cancer soon after. Its breakthrough designation can help speed this up.

But colorectal cancer patients don't have to wait until late 2024 to access Enhertu. In the meantime, what role could Enhertu play in your CRC treatment?

Can my doctor prescribe Enhertu for colorectal cancer?

The best answer currently is, maybe.

Enhertu is not approved for HER2-positive colorectal cancer treatment anywhere. However, your doctor has the authority to prescribe the medicine for this indication anyway. They could do this based on the final results of the DESTINY-CRC01 trial or the preliminary results of the DESTINY-CRC02 trial, and on the specifics of your case.

If a doctor gives you a medicine for a disease it's not approved for, that's off-label use. In some countries, filling an off-label use prescription may be challenging due to local regulations and availability. However, it's always possible to fill it using the Named Patient Import regulation.

Has your doctor made the decision to prescribe Enhertu for the treatment of your colorectal cancer? Our team at www.everyone.org can help you access the medicine. We specialize in sourcing and delivery of prescribed medicines unapproved or unavailable in a patient's country. Contact us, so we can help you.

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