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Lorlatinib: Mechanism, Dosage & Side Effects

Lorlatinib is a targeted cancer drug used to treat ALK- or ROS1-positive non-small cell lung cancer. It inhibits mutated kinases and effectively crosses the blood-brain barrier.

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Things worth knowing about "Lorlatinib"

Lorlatinib is a targeted cancer drug used to treat ALK- or ROS1-positive non-small cell lung cancer. It inhibits mutated kinases and effectively crosses the blood-brain barrier.

What is Lorlatinib?

Lorlatinib is an orally administered third-generation tyrosine kinase inhibitor (TKI) used in the treatment of non-small cell lung cancer (NSCLC) that carries a rearrangement of the ALK (Anaplastic Lymphoma Kinase) or ROS1 gene. These genetic alterations drive uncontrolled cancer cell growth, which Lorlatinib is specifically designed to block.

Indication

Lorlatinib is approved for adults with:

  • ALK-positive NSCLC as a first-line treatment or after prior therapy with another ALK inhibitor
  • ROS1-positive NSCLC following failure of earlier therapies (subject to national approval)

Lorlatinib is particularly valuable in patients with brain metastases, as the drug is capable of effectively crossing the blood-brain barrier and exerting its anti-tumor effect within the central nervous system.

Mechanism of Action

Lorlatinib selectively inhibits ALK and ROS1 kinase activity by binding to the active site of these enzymes. Compared to first- and second-generation TKIs (such as Crizotinib or Alectinib), Lorlatinib retains activity against a broad range of resistance mutations that can develop during prior therapy. Its unique molecular structure enables reliable penetration of the blood-brain barrier, providing effective anti-tumor activity within the central nervous system.

Dosage and Administration

Lorlatinib is taken orally as a film-coated tablet once daily. The standard recommended dose is 100 mg per day. The tablet can be taken with or without food. Dose reductions or temporary interruptions may be necessary in the event of significant side effects, as directed by the treating physician.

Side Effects

Like all cancer therapies, Lorlatinib may cause side effects. The most commonly reported include:

  • Edema (fluid retention, especially in the legs and arms)
  • Hypercholesterolemia and hypertriglyceridemia (elevated blood lipid levels)
  • Peripheral neuropathy (tingling or numbness in the hands and feet)
  • Cognitive effects (memory difficulties, mood changes, slowed thinking)
  • Hypertension (high blood pressure)
  • Weight gain

Patients should discuss any new or worsening symptoms with their doctor. Regular monitoring of blood lipid levels and neurological function is recommended throughout treatment.

Treatment Context

Lorlatinib is part of a targeted therapy approach for genetically defined tumors. Treatment is generally continued for as long as clinical benefit is observed and tolerability remains acceptable. Lorlatinib can be used following prior ALK inhibitors such as Crizotinib, Alectinib, or Brigatinib, as it retains potency against many of the resistance mutations that arise with these agents. In the first-line setting, the phase 3 CROWN trial demonstrated significantly improved progression-free survival with Lorlatinib compared to Crizotinib in patients with ALK-positive advanced NSCLC.

References

  1. European Medicines Agency (EMA): Lorbrena (Lorlatinib) - European Public Assessment Report, 2019. Available at: https://www.ema.europa.eu
  2. Shaw AT et al. - Lorlatinib in non-small-cell lung cancer with ALK or ROS1 rearrangement: an international, multicentre, open-label, single-arm first-in-man phase 1 trial. Lancet Oncol. 2017;18(12):1590-1599.
  3. Solomon BJ et al. - Lorlatinib versus Crizotinib in patients with advanced ALK-positive non-small-cell lung cancer (CROWN): 3-year follow-up of a randomised, open-label, phase 3 trial. Lancet Oncol. 2023;24(3):249-265.

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