Osimertinib: What Patients Need To Know
- A drug called osimertinib [brand name: Tagrisso] has transformed the outlook for patients with a specific type of lung cancer known as EGFR-positive, helping many people live longer after surgery.
- The medication is taken orally once a day after surgery and chemotherapy.
- It has also shown promise treating EGFR-positive patients even when surgery is not possible, extending progression-free survival.
- Side effects, such as diarrhea, skin rash, and tiredness, are common — but often controllable when they are reported early.
- “Osimertinib is a drug that really revolutionized the space for patients with non-small cell lung cancer,” Dr. Isabel Preeshagul, a thoracic medical oncologist at Memorial Sloan Kettering Cancer Center, tells SurvivorNet.
- It was originally approved in the stage IV setting, but has since been approved for earlier stage disease.
By blocking the overactive EGFR signal, the drug carries the potential to slow or stop cancer growth and, unlike many other EGFR drugs, can also cross the blood-brain barrier to tackle tumors or microscopic “seedlings” in the brain.
Read MoreAfter successful results treating stage IV lung cancer with the drug, researchers wanted to see if it could stop cancer progression in earlier stages. This approach was tested in the ADAURA trial.
Expanding Osimertinib’s Use
The ADAURA trial looked at patients that had resectable stage IB to stage III non-small cell lung cancer that harbored an EGFR alteration.
“These patients went on to get their resection and then chemotherapy. After that, they went on to receive three years of osimertinib. So these patients were randomized to either receive three years of osimertinib or a placebo (no medication). We found that patients that received osimertinib for three years after their surgery and after their chemo lived longer and had a longer amount of time without their cancer coming back. So this has become the new standard of care,” Dr. Preeshagul explains.
- After surgery for patients with stages IB–IIIA lung cancer, doctors may prescribe up to three years of osimertinib to lower the risk of the cancer recurrence (coming back).
- In the ADAURA study, 5-year overall survival was 85% with the pill versus 73% with placebo (no drug) — a 51 % reduction in the risk of death.
Dr. Preeshagul explains that in an additional trial, the LAURA trial, patients who had inoperable disease were given osimertinib. The promising results of this trial were published in June 2024.
“The separation of the curves here was tremendous,” Dr. Preeshagul explains of the data.
- For patients with unresectable (inoperable) disease who underwent chemoradiation followed by osimertinib, the drug can help prevent progression for far longer than observation alone.
- The LAURA trial reported a median progression-free survival (stable disease) of about 39 months on the drug compared with 5.6 months on placebo.
How Is Osimertinib Administered?
The usual dose is 80 mg once daily, swallowed whole at roughly the same time each day, with or without food.
The pharmaceutical recommendation is that if you miss a dose and fewer than twelve hours remain until your next one, skip it rather than doubling up.
Women and men who could conceive should use reliable birth control during therapy and for several weeks afterward, because the drug can be potentially dangerous for a fetus. Breastfeeding is not recommended during treatment or for two weeks after the final pill.
Side Effects: What To Expect
The presence of side effects is undeniable. However, most people present mild symptoms that are manageable with easy interventions. It is most common to notice side effects within the first month, although many become milder over time.
The most frequent and manageable effects include:
- Diarrhea
- Skin or acne-like rash
- Dry or itchy skin
- Brittle or tender nails
- Tiredness
- Mild aches.
The general recommendations to help avoid these issues include staying well-hydrated, starting an anti-diarrheal at the first sign of loose stools, using fragrance-free moisturizers, sun-protective clothing, and nail care.
“The most common [side effects] are rash, which is almost like an acne,” Dr. Preeshagul explains. “We often tell our patients ‘please wear sunscreen every day, stay out of the sun if you can, even when you’re driving, put the visor down if you’re going to go to the beach over the summer.'”
She adds that patients aren’t discouraged from living their lives and enjoying something like a planned vacation, for example, but it’s important for patients to be mindful. “Wear those long sleeve swimming suits and try to hang out under the umbrella if you can,” she suggests.
There are a few side effects of the treatment that could be severe and may warrant a trip to see your doctor or even to contact your local emergency department. These include:
- New or worsening cough
- Shortness of breath that could signal lung inflammation
- Fast or irregular heartbeat
- Ankle swelling
- Dizziness that may reflect heart-muscle weakness or rhythm changes
- Severe skin blistering or peeling
- Sudden eye pain or vision changes
“Some of the other more rare side effects would be cytopenias, which means bringing your blood counts down. Pneumonitis is another side effect that can happen, which is inflammation of the lungs,” Dr. Preeshagul explains.
“Another side effect of osimertinib is that it can affect your heart’s ability to pump. So we always want to get an ultrasound of your heart before you start this pill, and then it’s standard of care to get an ultrasound every three months to make sure that your heart’s pumping percentage does not decrease.”
It’s also important to note that patients should make all of their healthcare providers aware that they are taking osimertinib — including dentists and pharmacists. Certain stomach-acid reducers, tuberculosis antibiotics such as rifampin, and herbal products like St. John’s wort can lower drug levels in the blood.
Practical: What It Means for Patients
Chemotherapy plus osimertinib is emerging as a powerful first-line option, especially for people whose disease is widespread or rapidly growing.
The LAURA trial’s 2024 results made osimertinib the first targeted pill to improve outcomes after chemoradiation in unresectable stage III disease, reaching three years in average of cancer control, where six months had previously been the norm.
Patients who are eligible for this treatment should speak to their doctors about whether these evolving strategies — or enrolling in a newer clinical trial — are the right fit.
Questions To Ask Your Doctor
- Does my tumor carry an EGFR mutation that makes osimertinib a good choice?
- How often will we scan or test to be sure the medication is working?
- What monitoring will I need for my lungs, heart, and eyes?
- If the cancer grows in the future, what other options or trials can I try?
- Could any of my other prescriptions, over-the-counter medicines, or supplements interfere with osimertinib?
- Are there financial-assistance or patient-support programs that can help with the cost of this treatment?
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