Koselugo (Selumetinib)
MEK inhibitor for plexiform neurofibromas in neurofibromatosis type 1 (NF1)
Koselugo (selumetinib) is a targeted cancer therapy that works by inhibiting MEK1 and MEK2 enzymes. It is used to treat inoperable plexiform neurofibromas in patients with neurofibromatosis type 1 (NF1). The granule formulation is designed for young children (aged 1 to under 7 years) and patients who cannot swallow capsules. This guide covers dosage, side effects, drug interactions, and important safety information based on EMA and FDA prescribing data.
Quick Facts: Koselugo (Selumetinib)
Key Takeaways
- Koselugo (selumetinib) is the first approved targeted therapy for symptomatic, inoperable plexiform neurofibromas in NF1 patients.
- The granule formulation is sprinkled onto soft food and is designed for children aged 1 to under 7 years or patients who cannot swallow capsules.
- Regular monitoring of heart function (echocardiogram), eye examinations, and liver function tests is required during treatment.
- Common side effects include vomiting, diarrhea, skin/nail changes, fatigue, and decreased cardiac ejection fraction.
- Avoid grapefruit and Seville orange products during treatment as they can affect how the medicine works.
What Is Koselugo and What Is It Used For?
Quick Answer: Koselugo (selumetinib) is a MEK inhibitor used to treat plexiform neurofibromas that cannot be completely removed by surgery in patients with neurofibromatosis type 1 (NF1). The granule formulation is specifically designed for young children and patients unable to swallow capsules.
Koselugo contains the active substance selumetinib, which belongs to a class of medicines called MEK inhibitors. It works by blocking specific proteins (MEK1 and MEK2) that are involved in the growth and survival of tumor cells. In neurofibromatosis type 1, a genetic condition caused by mutations in the NF1 gene, the loss of the protein neurofibromin leads to overactivation of the RAS/MAPK signaling pathway. This uncontrolled signaling drives the growth of benign tumors called plexiform neurofibromas along nerves throughout the body.
Plexiform neurofibromas are complex tumors that grow along the length of nerves and can involve multiple branches and plexuses. Unlike other types of neurofibromas, plexiform neurofibromas can be large, disfiguring, and functionally debilitating. They may cause pain, motor deficits, airway compromise, visual impairment, and bladder or bowel dysfunction depending on their location. The tumors are often inoperable because of their diffuse infiltrating nature and proximity to vital structures.
By selectively inhibiting MEK1 and MEK2, Koselugo reduces the downstream signaling that drives tumor cell proliferation. In clinical trials (the SPRINT Stratum 1 study), selumetinib demonstrated the ability to shrink plexiform neurofibromas, with a confirmed partial response rate of approximately 66% in pediatric patients. Many patients also experienced meaningful improvements in pain, functional status, and quality of life.
Koselugo granules in capsules for opening are used for the treatment of symptomatic, inoperable plexiform neurofibromas in children aged 1 year to under 7 years with NF1, as well as in older patients who have difficulty swallowing hard capsules. The drug received conditional marketing authorization from the European Medicines Agency (EMA) in 2021 and FDA approval in the United States in 2020, making it the first pharmacological treatment specifically approved for this condition.
NF1 is one of the most common genetic disorders, affecting approximately 1 in 3,000 individuals worldwide. It is caused by mutations in the NF1 gene on chromosome 17, which encodes neurofibromin, a tumor suppressor protein. Plexiform neurofibromas develop in approximately 30-50% of NF1 patients and are present from birth, though they typically become symptomatic during childhood. Without treatment, these tumors can cause progressive morbidity and, in rare cases, may undergo malignant transformation to malignant peripheral nerve sheath tumors (MPNSTs).
What Should You Know Before Taking Koselugo?
Quick Answer: Do not take Koselugo if you are allergic to selumetinib or have severe liver disease. Your doctor must monitor your eyes, heart function, and liver enzymes regularly. Koselugo is not recommended during pregnancy or breastfeeding.
Contraindications
Koselugo must not be taken in the following situations:
- Allergy to selumetinib or any of the other ingredients in the formulation (glycerol dibehenate, stearoyl macrogol glycerides, hypromellose acetate succinate, stearic acid, titanium dioxide, iron oxides)
- Severe liver disease (severe hepatic impairment), as the drug is primarily metabolized by the liver and exposure may be significantly increased
If either of these conditions applies, inform your healthcare provider before taking Koselugo. Treatment should not be initiated until the healthcare provider has confirmed it is safe to proceed.
Warnings and Precautions
Before and during treatment with Koselugo, you should discuss the following important warnings with your doctor, pharmacist, or nurse:
Koselugo can cause ocular toxicities including serous retinal detachment and retinal vein occlusion. Contact your doctor immediately if you experience blurred vision or any other changes in vision during treatment. Your doctor should examine your eyes if you develop any new or worsening visual symptoms. Ophthalmological examinations should be performed at baseline and periodically during treatment. Treatment may need to be interrupted or discontinued depending on the severity of ocular findings.
Koselugo can decrease the amount of blood your heart pumps (decreased left ventricular ejection fraction, or LVEF). Your doctor will check your heart function with an echocardiogram before starting treatment and at regular intervals during therapy. If a significant decrease in LVEF is detected, your dose may be reduced, treatment temporarily interrupted, or permanently discontinued. Patients with pre-existing cardiac conditions should be monitored more closely.
Koselugo can increase the levels of certain liver enzymes (ALT, AST, and GGT) in the blood, indicating hepatotoxicity. Your doctor will perform blood tests before starting treatment and regularly during therapy to monitor liver function. If significant elevations are detected, your dose may need to be adjusted or treatment interrupted. Patients with pre-existing liver disease or impairment require closer monitoring and may need dose modifications.
Additional precautions to be aware of during treatment:
- Skin, nail, and hair problems: Koselugo frequently causes dermatological reactions including skin rash, dry skin, acne-like eruptions, nail infections (paronychia), hair thinning (alopecia), and changes in hair color. While these are generally manageable, inform your doctor if any of these symptoms become bothersome or severe. Proactive dermatological care including moisturizers and sun protection is recommended.
- High blood pressure: Hypertension has been reported with Koselugo. Blood pressure should be monitored regularly and treated with antihypertensive medications as needed.
- Gastrointestinal effects: Vomiting, nausea, and diarrhea are very common. Adequate hydration and anti-emetic or anti-diarrheal medications may be necessary.
Use in Children Under 1 Year
Koselugo granules should not be given to children under 1 year of age. The safety and efficacy of selumetinib have not been established in this age group, and there is insufficient data to recommend its use. Clinical trials enrolled patients from 1 year of age and older.
Pregnancy and Breastfeeding
Koselugo is not recommended during pregnancy. Based on its mechanism of action and findings from animal studies, selumetinib may cause harm to an unborn baby. If you think you may be pregnant or are planning to have a baby, consult your doctor before starting this medicine. A pregnancy test may be required before treatment begins.
Contraception: If you are sexually active, you must use effective contraception during treatment and for at least 1 week after the last dose. It is not known whether Koselugo affects how well hormonal contraceptives work. If you are using hormonal contraception, your doctor may recommend adding a non-hormonal method as well. Male patients whose partners are or may become pregnant should also use effective contraception during treatment and for at least 1 week after the last dose.
Breastfeeding: Do not breastfeed while taking Koselugo. It is not known whether selumetinib or its metabolites pass into breast milk. Because of the potential for serious adverse reactions in a breastfed infant, breastfeeding should be discontinued during treatment and for at least 1 week after the last dose.
Driving and Operating Machinery
Koselugo may cause side effects that affect your ability to drive or operate machinery. Do not drive or use machines if you feel tired or if you have vision problems such as blurred vision. If you are affected, wait until these symptoms resolve before driving or operating machinery.
How Does Koselugo Interact with Other Drugs?
Quick Answer: Koselugo interacts with many common medications including antibiotics (clarithromycin, erythromycin), antifungals (fluconazole, itraconazole, ketoconazole), anti-epileptics (carbamazepine, phenytoin), and others. Always inform your doctor about all medicines you are taking, including herbal remedies and supplements.
Selumetinib is metabolized primarily by CYP3A4 and CYP1A2 enzymes and is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein (BCRP). Drugs that inhibit or induce these enzymes or transporters can significantly alter selumetinib exposure, potentially leading to increased toxicity or reduced efficacy. Similarly, selumetinib may affect the metabolism or transport of other drugs.
Major Interactions
The following drug interactions are considered clinically significant and may require dose adjustments, increased monitoring, or avoidance of concomitant use:
| Drug | Used For | Type of Interaction | Clinical Effect |
|---|---|---|---|
| Clarithromycin | Bacterial infections | Strong CYP3A4 inhibitor | May significantly increase selumetinib levels, increasing risk of side effects |
| Itraconazole | Fungal infections | Strong CYP3A4 inhibitor | May significantly increase selumetinib levels, increasing risk of side effects |
| Ketoconazole | Cushing's syndrome | Strong CYP3A4 inhibitor | May significantly increase selumetinib levels, increasing risk of side effects |
| Rifampicin | Tuberculosis, bacterial infections | Strong CYP3A4 inducer | May significantly decrease selumetinib levels, reducing efficacy |
| Carbamazepine | Seizures, epilepsy | Strong CYP3A4 inducer | May significantly decrease selumetinib levels, reducing efficacy |
| Phenytoin | Seizures, epilepsy | Strong CYP3A4 inducer | May significantly decrease selumetinib levels, reducing efficacy |
| St. John's Wort | Mild depression (herbal remedy) | CYP3A4 inducer | May decrease selumetinib levels, reducing efficacy |
Other Notable Interactions
The following medications may also interact with Koselugo and should be discussed with your healthcare provider:
| Drug | Used For | Interaction Mechanism |
|---|---|---|
| Erythromycin | Bacterial infections | Moderate CYP3A4 inhibitor; may increase selumetinib exposure |
| Fluconazole | Fungal infections | Moderate CYP3A4 inhibitor; may increase selumetinib exposure |
| Digoxin | Heart failure | P-gp substrate; selumetinib may increase digoxin levels |
| Fexofenadine | Allergic symptoms | P-gp substrate; selumetinib may affect fexofenadine levels |
| Furosemide | Fluid retention (diuretic) | Transporter interaction; may affect furosemide elimination |
| Methotrexate | Cancer, psoriasis, rheumatoid arthritis | BCRP substrate; selumetinib may increase methotrexate exposure |
| Omeprazole | Acid reflux, stomach ulcers | CYP interaction; may affect selumetinib metabolism |
| Ticlopidine | Blood clot prevention | CYP interaction; may affect selumetinib metabolism |
Food Interactions
Koselugo granules are given together with food, but certain foods can affect how the medicine works. Important dietary precautions include:
- Grapefruit and grapefruit juice: Must be avoided during treatment. Grapefruit inhibits CYP3A4 in the gut and can increase selumetinib absorption, leading to higher blood levels and increased risk of side effects.
- Seville (bitter) oranges: Products containing Seville orange juice, puree, or jam should be avoided as they contain similar CYP3A4 inhibitors.
- Do not mix with: Water, milk, or vegetable puree. These may affect the stability or absorption of the granules.
- Suitable foods: Plain yogurt, fruit puree (not grapefruit or Seville orange), fruit jam (not grapefruit or Seville orange), or similar soft foods.
What Is the Correct Dosage of Koselugo?
Quick Answer: The dose of Koselugo is calculated based on body surface area (BSA) and is taken twice daily, approximately 12 hours apart. The granules are sprinkled on soft food and must be consumed within 30 minutes. Your doctor will determine the correct dose for your specific situation.
Koselugo dosing is individualized based on body surface area (BSA), which is calculated from the patient's height and weight. The recommended dose for patients with normal liver function is 25 mg/m2 twice daily. The doctor will calculate the appropriate combination of 5 mg and 7.5 mg capsules needed to achieve the target dose.
Children (1 Year to Under 7 Years)
Pediatric Dosing (Granules)
The recommended dose is 25 mg/m2 twice daily, approximately 12 hours apart. The capsules should be opened and the granules sprinkled onto a small amount (approximately 1 to 3 teaspoons) of soft food. The exact number of capsules and their strengths will be determined by the prescribing physician based on the child's BSA.
Koselugo hard capsules (10 mg, 25 mg) are also available and may be prescribed for children aged 3 years and older who can swallow capsules whole.
Older Patients Unable to Swallow Capsules
Granule Formulation for Swallowing Difficulties
For patients of any age who have difficulty swallowing, the granule formulation can be used at the same BSA-based dose of 25 mg/m2 twice daily. The preparation and administration method is identical to that for pediatric patients.
Patients with Liver Impairment
Hepatic Impairment
Patients with mild hepatic impairment (Child-Pugh A) do not require dose adjustment. Patients with moderate hepatic impairment (Child-Pugh B) may require a lower dose as determined by the prescribing physician. Koselugo is contraindicated in patients with severe hepatic impairment (Child-Pugh C).
Dose Adjustments for Side Effects
Your doctor may reduce the dose, temporarily interrupt treatment, or permanently discontinue Koselugo if you experience certain side effects. Common reasons for dose modification include:
- Decreased cardiac ejection fraction (heart pumping efficiency)
- Significant elevation of liver enzymes
- Severe ocular toxicity (eye problems)
- Intolerable skin reactions
- Persistent severe gastrointestinal symptoms
Do not change your dose or stop taking Koselugo without consulting your doctor. Abrupt discontinuation should be avoided unless medically advised.
How to Prepare and Take Koselugo Granules
Follow these steps carefully each time you take or give Koselugo granules:
- Wash and dry hands thoroughly. Open the container and take out the correct number of capsules of the correct color for the prescribed dose.
- Prepare soft food. Place a small amount (approximately 1 to 3 teaspoons) of soft food such as plain yogurt, fruit puree, or fruit jam onto a spoon or into a small cup.
- Open capsules carefully. Open one capsule at a time and sprinkle the entire contents (granules) over the soft food. Do not swallow, chew, or dissolve the capsule shells. Do not mix the granules with water, milk, vegetable puree, or any grapefruit or Seville orange products.
- Swallow the mixture. Sit upright and swallow the mixture of granules and food without chewing, within 30 minutes of preparation. Do not save for later use.
- Ensure complete dose. After swallowing, add a small amount of the chosen food to the cup to collect any remaining granules and swallow. Discard the empty capsule shells.
Once the granules are sprinkled onto food, the mixture must be swallowed within 30 minutes. If not taken within this time, the dose should be discarded and a new dose prepared at the next scheduled time. If only part of the dose was taken within 30 minutes, discard the remainder and do not prepare a new dose. Aim to take the entire dose within the 30-minute window next time.
Missed Dose
What to do if you miss a dose depends on when you remember:
- More than 6 hours until next dose: Take the missed dose as soon as you remember. Then take the next dose at the usual time.
- Less than 6 hours until next dose: Skip the missed dose entirely. Take the next dose at the usual time.
Do not take a double dose (two doses at the same time) to make up for a missed dose.
Overdose
If you or someone else takes more Koselugo than prescribed, contact your doctor or seek emergency medical attention immediately. Symptoms of overdose may include increased severity of known side effects such as nausea, vomiting, diarrhea, skin rash, and visual disturbances. There is no specific antidote for selumetinib overdose; treatment is supportive and symptomatic.
If You Vomit After Taking Koselugo
If you vomit after taking a dose of Koselugo, regardless of when the vomiting occurs, do not take an additional dose. Simply take the next dose at the usual scheduled time. This applies whether the vomiting occurs immediately after taking the medicine or several hours later.
What Are the Side Effects of Koselugo?
Quick Answer: The most common side effects of Koselugo include vomiting, nausea, diarrhea, mouth inflammation, skin and nail problems, hair changes, fatigue, fever, peripheral edema, decreased cardiac ejection fraction, and high blood pressure. Blurred vision is a less common but potentially serious side effect requiring immediate medical attention.
Like all medicines, Koselugo can cause side effects, although not everybody gets them. The side effects listed below have been reported in clinical trials in both pediatric and adult patients. The frequency categories are defined as: very common (affects more than 1 in 10 people), common (affects 1 to 10 in 100 people), uncommon (affects 1 to 10 in 1,000 people), and rare (affects fewer than 1 in 1,000 people).
Serious Side Effects Requiring Immediate Attention
Contact your doctor immediately if you experience any of the following visual symptoms, which may indicate serous retinal detachment or other ocular toxicity:
- Blurred vision (common, affecting up to 1 in 10 people)
- Loss of vision
- Dark spots in your field of vision (floaters)
- Any other changes in vision (such as reduced visual acuity)
Your doctor may need to discontinue treatment or refer you to an ophthalmologist.
Side Effect Frequency in Pediatric Patients
Very Common
Affects more than 1 in 10 people
- Vomiting, nausea
- Diarrhea
- Mouth inflammation (stomatitis)
- Skin and nail problems (dry skin, rash, redness around fingernails, acne-like rash, inflammation of hair follicles)
- Hair thinning (alopecia), changes in hair color
- Tiredness, weakness, or lack of energy
- Fever (pyrexia)
- Swelling of hands or feet (peripheral edema)
- Decreased cardiac ejection fraction (reduced heart pumping efficiency)
- High blood pressure (hypertension)
- Decreased albumin in the blood (shown in blood tests)
- Decreased hemoglobin (shown in blood tests)
- Elevated liver enzymes, creatine phosphokinase (shown in blood tests)
Common
Affects 1 to 10 in 100 people
- Dry mouth
- Facial swelling (facial edema)
- Shortness of breath or breathlessness (dyspnea)
Side Effects in Adult Patients
The following side effects have been reported in clinical studies with adult patients receiving Koselugo. The side effect profile is broadly similar to that seen in pediatric patients, with some differences in frequency:
Very Common (Adults)
Affects more than 1 in 10 people
- Vomiting, nausea
- Diarrhea, constipation
- Mouth inflammation (stomatitis)
- Skin and nail problems (dry skin, rash, acne-like rash, nail infections)
- Hair thinning (alopecia), changes in hair color
- Tiredness, weakness, or lack of energy
- Swelling of hands or feet (peripheral edema)
- Decreased hemoglobin (shown in blood tests)
- Elevated liver and kidney enzymes (shown in blood tests)
Common (Adults)
Affects 1 to 10 in 100 people
- Dry mouth
- Fever (pyrexia)
- Facial swelling (facial edema)
- Shortness of breath (dyspnea)
- Decreased albumin (shown in blood tests)
- Decreased cardiac ejection fraction
- High blood pressure (hypertension)
- Elevated creatine phosphokinase indicating muscle breakdown (shown in blood tests)
Reporting Side Effects
If you experience any side effects, including those not listed above, talk to your doctor, pharmacist, or nurse. You can also report side effects directly to your national medicines regulatory authority. By reporting side effects, you contribute to the ongoing monitoring of the medicine's safety profile. In the EU, side effects can be reported through national reporting systems, and in the US, through the FDA MedWatch program.
How Should You Store Koselugo?
Quick Answer: Store Koselugo below 25°C (77°F) in the original container, tightly closed, away from moisture and light. Keep out of reach of children. Do not remove the desiccant. Do not use after the expiry date.
Proper storage of Koselugo is essential to maintain the effectiveness and safety of the medicine. Follow these storage instructions carefully:
- Temperature: Store at or below 25°C (77°F). Do not freeze.
- Container: Keep in the original white plastic container with child-resistant closure. The container is designed to protect the granules from moisture and light.
- Desiccant: The container includes a silica gel desiccant. Do not remove the desiccant from the container and do not swallow it. It helps absorb moisture and maintain the stability of the granules.
- Closure: Always close the container tightly after each use to protect the remaining capsules from moisture and light exposure.
- Expiry date: Do not use Koselugo after the expiry date printed on the container and carton after "EXP." The expiry date refers to the last day of the stated month.
- Out of reach: Store this medicine out of the sight and reach of children.
Do not dispose of medicines via wastewater or household waste. Ask your pharmacist how to properly dispose of medicines that are no longer needed. These measures help protect the environment and prevent accidental exposure.
What Does Koselugo Contain?
Quick Answer: Koselugo contains selumetinib (as hydrogen sulfate) as the active ingredient. The granules contain excipients including glycerol dibehenate and hypromellose acetate succinate. The capsule shells are made of hypromellose and are color-coded: yellow for 5 mg and pink for 7.5 mg.
Active Ingredient
The active substance in Koselugo is selumetinib, present as selumetinib hydrogen sulfate:
- Koselugo 5 mg: Each capsule for opening contains 5 mg of selumetinib (as hydrogen sulfate)
- Koselugo 7.5 mg: Each capsule for opening contains 7.5 mg of selumetinib (as hydrogen sulfate)
Inactive Ingredients (Excipients)
The granules contain the following inactive ingredients: glycerol dibehenate, stearoyl macrogol glycerides, hypromellose acetate succinate, and stearic acid.
The capsule shell ingredients differ slightly between strengths:
- 5 mg capsule shell: Hypromellose (E464), titanium dioxide (E171), yellow iron oxide (E172), and printing ink
- 7.5 mg capsule shell: Hypromellose (E464), titanium dioxide (E171), red iron oxide (E172), and printing ink
The printing ink on both strengths contains: shellac (E904), propylene glycol (E1520), concentrated ammonia solution (E527), black iron oxide (E172), and potassium hydroxide (E525).
Appearance and Packaging
Koselugo is supplied as hard capsules designed to be opened. The two strengths are visually distinct to help prevent dosing errors:
- 5 mg: Yellow cap and white body. The cap is marked with "sel 5" and the body shows an image of a capsule with granules to indicate it is meant to be opened.
- 7.5 mg: Pink cap and white body. The cap is marked with "sel 7.5" and the body shows an image of a capsule with granules to indicate it is meant to be opened.
Each container holds 60 capsules and includes a silica gel desiccant. The container is a white plastic bottle with a child-resistant cap.
Frequently Asked Questions About Koselugo
Koselugo (selumetinib) is used to treat plexiform neurofibromas that cannot be completely removed by surgery in patients with neurofibromatosis type 1 (NF1). The granule formulation is specifically designed for children aged 1 year to under 7 years and for older patients who have difficulty swallowing capsules. It is the first targeted therapy approved for this specific indication and works by blocking MEK1 and MEK2 proteins that drive tumor growth.
The most common side effects (affecting more than 1 in 10 people) include vomiting, nausea, diarrhea, mouth inflammation, skin problems (dry skin, rash, acne-like eruptions), nail problems, hair thinning or color changes, tiredness, fever, swelling of hands or feet, decreased cardiac ejection fraction, high blood pressure, and changes in blood test results (decreased albumin and hemoglobin, elevated liver enzymes). Most of these side effects are manageable with supportive care and dose adjustments.
Open the capsules carefully and sprinkle the granules over a small amount (1 to 3 teaspoons) of soft food such as plain yogurt, fruit puree, or fruit jam. Do not swallow the capsule shells. Do not mix with water, milk, vegetable puree, or any grapefruit or Seville orange products. Swallow the mixture within 30 minutes of preparation without chewing. Take twice daily, approximately 12 hours apart. After swallowing, add more food to the cup to collect and swallow any remaining granules.
No. Koselugo is not recommended during pregnancy as it may cause harm to the unborn baby. Women who could become pregnant must use effective contraception during treatment and for at least 1 week after the last dose. Breastfeeding should be discontinued during treatment and for at least 1 week after the last dose, as it is not known whether selumetinib passes into breast milk. A pregnancy test may be required before starting treatment.
Regular monitoring is essential during Koselugo treatment. Your doctor will perform: echocardiograms to assess heart function (left ventricular ejection fraction) before starting and periodically during treatment; blood tests to monitor liver enzyme levels (ALT, AST, GGT) and other laboratory parameters; blood pressure measurements at regular intervals; and ophthalmological (eye) examinations at baseline and periodically during treatment, especially if any visual symptoms develop. The frequency of monitoring may be adjusted based on your individual response to treatment.
If you miss a dose and there are more than 6 hours until your next dose, take the missed dose right away and then take the next dose at the usual time. If there are fewer than 6 hours until the next dose, skip the missed dose and take the next one at the normal time. Never take a double dose. If you vomit after taking Koselugo (regardless of timing), do not take an additional dose. Simply wait and take the next dose at the scheduled time.
References
This article is based on the following peer-reviewed sources and regulatory documents:
- European Medicines Agency (EMA). Koselugo (selumetinib) Summary of Product Characteristics. Last updated January 2026. Available from: EMA - Koselugo.
- U.S. Food and Drug Administration (FDA). Koselugo (selumetinib) Prescribing Information. Revised 2024. AstraZeneca Pharmaceuticals LP.
- Gross AM, Wolters PL, Dombi E, et al. Selumetinib in Children with Inoperable Plexiform Neurofibromas. New England Journal of Medicine. 2020;382(15):1430-1442. doi:10.1056/NEJMoa1912735.
- Dombi E, Baldwin A, Marcus LJ, et al. Activity of Selumetinib in Neurofibromatosis Type 1-Related Plexiform Neurofibromas. New England Journal of Medicine. 2016;375(26):2550-2560. doi:10.1056/NEJMoa1605943.
- Gutmann DH, Ferner RE, Listernick RH, et al. Neurofibromatosis type 1. Nature Reviews Disease Primers. 2017;3:17004. doi:10.1038/nrdp.2017.4.
- World Health Organization (WHO). WHO Model List of Essential Medicines for Children. 8th list, 2023.
- British National Formulary (BNF). Selumetinib. NICE Evidence Services. Accessed January 2026.
- National Comprehensive Cancer Network (NCCN). Central Nervous System Cancers - Neurofibromatosis Type 1 Associated Tumors. Version 1.2025.
Editorial Team
This article has been reviewed by the iMedic Medical Review Board, an independent panel of physicians with expertise in oncology, pharmacology, and pediatric medicine.
Written by qualified medical professionals with expertise in oncology and pharmacology, following international guidelines from EMA, FDA, WHO, and BNF.
All medical claims are supported by Level 1A evidence from systematic reviews, randomized controlled trials, and international regulatory assessments (GRADE framework).
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