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Melanoma targeted therapy

Targeted Therapy for Melanoma: — BRAF and MEK Inhibitors

Targeted therapy for melanoma depends on a specific mutation in your tumour. A BRAF V600 mutation makes you eligible for oral combination tablets that directly block the pathway driving your cancer. The first step is testing the tumour tissue — not all melanomas carry this mutation.

Medically reviewed by Dr. Bharati Devi Gorantla, Medical Oncologist, MBBS · MD · DM (Adyar, Chennai) · ECMO · MRCP SCE (UK) · Last reviewed August 2026

  • Not all melanomas qualify — Only melanomas with a confirmed BRAF V600 mutation respond to this class of treatment. Testing comes before any treatment decision.
  • Oral tablets, not infusions — BRAF and MEK inhibitors are taken as daily tablets at home, not given by drip in hospital.
  • Usually given together — A BRAF inhibitor and a MEK inhibitor are combined because the combination works better and delays resistance longer than either alone.
  • Resistance is a known issue — Most melanomas eventually find a way around this treatment. Your team plans for that from the start.
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Targeted therapy for melanoma works when your tumour carries a BRAF V600 mutation. Your oncologist will test for this before recommending treatment. Those who carry the mutation are offered a combination of a BRAF inhibitor and a MEK inhibitor, taken as daily oral tablets.

Which mutations does your doctor test for in melanoma?

BRAF mutation testing is recommended for all patients with newly diagnosed advanced or metastatic cutaneous melanoma, according to NCCN and ESMO guidance. The most important variants are BRAF V600E and BRAF V600K. Either result makes the cancer eligible for BRAF-targeted treatment.

NRAS mutations are found in a smaller proportion of melanomas. There are currently fewer approved targeted options for NRAS-mutant melanoma, and your oncologist will explain what those are if your result comes back positive.

C-KIT mutations are seen mainly in acral melanoma — the type that appears on palms, soles, or under nails — and in mucosal melanoma. Targeted drugs that act on C-KIT exist, though the evidence base differs from the BRAF pathway.

Uveal melanoma, which starts in the eye, has a different molecular biology and does not carry BRAF V600 mutations. If you have uveal melanoma, BRAF and MEK inhibitors do not apply to your situation.

Testing is done on a sample of your tumour tissue, usually from the biopsy you have already had. Results typically take one to two weeks.

What happens from biopsy to starting treatment?

  1. Tumour tissue is sent for molecular testing

    The biopsy sample you have already had is usually enough. The laboratory analyses the DNA for BRAF and other mutations. No new procedure is generally needed at this stage.

  2. Your oncologist reviews the results with you

    If a BRAF V600 mutation is present, your oncologist will explain which combination of inhibitors is appropriate and what to expect. If no mutation is found, they will outline what the other options are.

  3. Baseline assessments are done before you start

    Blood tests and scans check the extent of the disease and your overall fitness. These results guide which combination and dose is right for you.

  4. You begin taking tablets at home

    BRAF and MEK inhibitors are oral tablets. Your team will tell you the schedule — usually once or twice daily — and what to take them with. There are no daily infusion visits.

  5. Your team monitors you at regular intervals

    Blood tests and scans happen at set points to check whether the treatment is working and to catch side effects early. Tell your team about any new symptom between appointments rather than waiting.

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What does treatment feel like day to day?

Most people take their tablets at home and continue with normal daily life between check-ups. The biggest practical difference from chemotherapy is that there are no weekly infusion visits.

Fatigue is common, particularly in the first weeks. A raised temperature — sometimes called treatment-related fever — is a side effect linked to one class of BRAF inhibitor. Your team will tell you what temperature level means calling them the same day.

Skin changes are frequent: rash, dryness, sensitivity to sunlight, and occasionally new skin lesions. Your oncologist will examine your skin at regular appointments because some of these lesions need to be checked.

Joint and muscle aches, nausea, and headache are also reported. Most are manageable and settle as your body adjusts. Report any new or worsening symptom rather than assuming it is expected.

Tell your team the same day if you notice any of these

  • A fever or a temperature that feels higher than your normal
  • A new rash that is spreading, blistering, or painful
  • Eye pain, redness, or any sudden change in vision
  • Chest pain or shortness of breath at rest
  • Severe joint or muscle pain that stops you moving normally
  • Yellowing of the skin or eyes, or very dark urine
  • A new lump or skin lesion that appears while you are on treatment
  • Any side effect that makes you want to stop taking your tablets on your own

What if your melanoma does not have a BRAF mutation?

Not having a BRAF mutation does not mean you are out of options. For most patients with advanced melanoma who are not BRAF-positive, immunotherapy — checkpoint inhibitors — is the standard first-line approach recommended by NCCN and ESMO.

If your melanoma has an NRAS or C-KIT mutation, your oncologist will explain what targeted or clinical trial options exist for those specific findings.

It is also possible to lose response to BRAF and MEK inhibitors over time even if they worked well initially. When that happens, your team will review the options — which may include immunotherapy, a different drug combination, or a clinical trial.

Ask your oncologist to explain your mutation result plainly and what treatment pathway it leads to. A result that rules out one treatment is also a result that points clearly toward another.

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Common questions

Frequently asked questions

How long will I need to take these tablets?

Treatment continues for as long as it is working and the side effects are manageable. There is no fixed course length the way there is with chemotherapy cycles. Most melanomas eventually develop resistance to BRAF and MEK inhibitors, and your oncologist plans for that possibility from the start. Scans at regular intervals show whether the treatment is still controlling the disease. If it stops working, your team will discuss what comes next, which often includes immunotherapy.

Can BRAF and MEK inhibitors be combined with immunotherapy?

In some situations, yes, though the evidence on combining all three at once is still developing. NCCN and ESMO guidance focuses primarily on using a BRAF inhibitor and a MEK inhibitor together as a pair, and on sequencing them with immunotherapy rather than giving everything simultaneously. Whether combination or sequencing is right for you depends on your stage, fitness, and how the disease is behaving. Your oncologist will explain the reasoning behind the order they recommend.

Is targeted therapy for melanoma available at CION?

Yes. BRAF and MEK inhibitor treatment is administered as day care at CION centres, which means no overnight stay is needed for most appointments. Imaging used to assess your response — including PET-CT — is coordinated with partner imaging centres. Your oncologist will confirm which combination is appropriate once your biomarker results are available.

Can I take herbal supplements or other medicines alongside these tablets?

Tell your oncology team before taking anything new — including herbal preparations and medicines bought without a prescription. Some interact with BRAF and MEK inhibitors in ways that reduce how well they work or increase side effects. This is a check to keep the treatment working as intended, not a blanket restriction on everything. If your team has already told you a particular medicine is fine for you, follow that advice.

Do these drugs work for all types of melanoma?

No. BRAF and MEK inhibitors apply only to cutaneous melanoma — the type that starts in the skin — with a confirmed BRAF V600 mutation. They are not used for uveal melanoma, which starts in the eye and has different molecular biology. Acral and mucosal melanomas carry different mutation profiles and are managed differently. Your oncologist will tell you which type you have and what treatment pathway that leads to.

What should I ask at my next appointment?

Ask three specific things: what your BRAF mutation result was and what it means for your treatment options; if you are already on treatment, what your next scan will tell the team and when it is scheduled; and what the plan is if the current treatment stops working. Writing the answers down during the appointment helps, because these conversations are hard to recall afterwards. It is entirely reasonable to ask for the mutation result in writing.

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