Medical Dialogues
  • Dermatology
Sign in Signup
This site is intended for healthcare professionals only
Sign in Signup
  • Medical Jobs
  • Medical Matrimony
  • MD Brand Connect
  • MDTV
Medical Dialogues
  • Medical News
      • Anesthesiology
      • Cardiology and CTVS
      • Critical Care
      • Dentistry
      • Dermatology
      • Diabetes and Endocrinology
      • ENT
      • Gastroenterology
      • Medicine
      • Nephrology
      • Neurology
      • Obstretics-Gynaecology
      • Oncology
      • Ophthalmology
      • Orthopaedics
      • Pediatrics-Neonatology
      • Psychiatry
      • Pulmonology
      • Radiology
      • Surgery
      • Urology
      • Laboratory Medicine
      • Diet
      • Nursing
      • Paramedical
      • Physiotherapy
  • Health news
      • Doctor News
      • Government Policies
      • Hospital & Diagnostics
      • International Health News
      • MCI News
      • Medical Organization News
      • Medico Legal News
      • NBE News
      • NMC News
  • Medical Guidelines
      • Anesthesiology
      • Cardiology and CTVS
      • Critical Care
      • Dentistry
      • Dermatology
      • Diabetes and Endocrinology
      • ENT
      • Gastroenterology
      • Medicine
      • Nephrology
      • Neurology
      • Obstretics-Gynaecology
      • Oncology
      • Ophthalmology
      • Orthopaedics
      • Pediatrics-Neonatology
      • Psychiatry
      • Pulmonology
      • Radiology
      • Surgery
      • Urology
      • Laboratory Medicine
      • Diet
      • Nursing
      • Paramedical
      • Physiotherapy
  • AYUSH
    • Ayurveda
    • Homeopathy
    • Siddha
    • Unani
    • Yoga
  • State News
      • Andaman and Nicobar Islands
      • Andhra Pradesh
      • Arunachal Pradesh
      • Assam
      • Bihar
      • Chandigarh
      • Chattisgarh
      • Dadra and Nagar Haveli
      • Daman and Diu
      • Delhi
      • Goa
      • Gujarat
      • Haryana
      • Himachal Pradesh
      • Jammu & Kashmir
      • Jharkhand
      • Karnataka
      • Kerala
      • Ladakh
      • Lakshadweep
      • Madhya Pradesh
      • Maharashtra
      • Manipur
      • Meghalaya
      • Mizoram
      • Nagaland
      • Odisha
      • Puducherry
      • Punjab
      • Rajasthan
      • Sikkim
      • Tamil Nadu
      • Telangana
      • Tripura
      • Uttar Pradesh
      • Uttrakhand
      • West Bengal
  • Medical Education
      • Ayush Education News
      • Dentistry Education News
      • Medical Admission News
      • Medical Colleges News
      • Medical Courses News
      • Medical Universities News
      • Nursing education News
      • Paramedical Education News
      • Study Aborad
  • Industry
    • Health Investment News
    • Health Startup News
    • Medical Devices News
    • Pharma News
  • MDTV
      • Anesthesia
      • CTVS
      • Cardiology
      • Critical Care
      • Dentistry
      • Dermatology
      • Diabetes and Endocrinology
      • Diet and nutrition
      • ENT
      • Gastroenterology
      • Health Dialogues
      • Health News today
      • Health Shorts
      • Health Updates
      • Health video of the day
      • Laboratory Medicine
      • Latest Videos
      • Latest Webinars
      • MD shorts
      • Medical News Today
      • Medical Updates
      • Medical Video of the day
      • Medicine
      • Nephrology
      • Neurology and Neurosurgery
      • Obstetrics and Gynaecology
      • Oncology
      • Ophthalmology
      • Orthopaedics
      • Pediatrics & Neonatology
      • Psychiatry
      • Pulmonology
      • Radiology
      • Surgery
      • Top Videos
      • Urology
This site is intended for healthcare professionals only
Sign InRegister
Medical Dialogues
Sign inRegister
  • Home
  • Medical news
    • Anesthesiology
    • Cardiology and CTVS
    • Critical Care
    • Dentistry
    • Dermatology
    • Diabetes and Endocrinology
    • ENT
    • Gastroenterology
    • Medicine
    • Nephrology
    • Neurology
    • Obstretics-Gynaecology
    • Oncology
    • Ophthalmology
    • Orthopaedics
    • Pediatrics-Neonatology
    • Psychiatry
    • Pulmonology
    • Radiology
    • Surgery
    • Urology
    • Laboratory Medicine
    • Diet
    • Nursing
    • Paramedical
    • Physiotherapy
  • Health news
    • Doctor News
    • Government Policies
    • Hospital & Diagnostics
    • International Health News
    • MCI News
    • Medical Organization News
    • Medico Legal News
    • NBE News
    • NMC News
  • Medical Guidelines
    • Anesthesiology
    • Cardiology and CTVS
    • Critical Care
    • Dentistry
    • Dermatology
    • Diabetes and Endocrinology
    • ENT
    • Gastroenterology
    • Medicine
    • Nephrology
    • Neurology
    • Obstretics-Gynaecology
    • Oncology
    • Ophthalmology
    • Orthopaedics
    • Pediatrics-Neonatology
    • Psychiatry
    • Pulmonology
    • Radiology
    • Surgery
    • Urology
    • Laboratory Medicine
    • Diet
    • Nursing
    • Paramedical
    • Physiotherapy
  • Medical Education
    • Ayush Education News
    • Dentistry Education News
    • Medical Admission News
    • Medical Colleges News
    • Medical Courses News
    • Medical Universities News
    • Nursing education News
    • Paramedical Education News
    • Study Aborad
  • Industry
    • Health Investment News
    • Health Startup News
    • Medical Devices News
    • Pharma News
      • CDSCO (Central Drugs Standard Control Organisation) News
  • Case of the Day
  • Editorial
  • Home
  • Pediatrics and Neonatology
  • Pediatrics and Neonatology News
  • FDA approves first...

FDA approves first treatment for Neurofibromatosis in children

Dr. Kamal Kant KohliBy Dr. Kamal Kant KohliPublished On 2020-04-11T07:08:09+05:30  |  Updated On 2020-04-11T07:54:55+05:30
FDA approves first treatment for Neurofibromatosis in children

The U.S. Food and Drug Administration has approved Koselugo (selumetinib) for the treatment of pediatric patients, 2 years of age and older, with neurofibromatosis type 1 (NF1), a genetic disorder of the nervous system causing tumors to grow on nerves. Koselugo is the first drug approved by the FDA to treat this debilitating, progressive and often disfiguring rare disease that typically...

The U.S. Food and Drug Administration has approved Koselugo (selumetinib) for the treatment of pediatric patients, 2 years of age and older, with neurofibromatosis type 1 (NF1), a genetic disorder of the nervous system causing tumors to grow on nerves. Koselugo is the first drug approved by the FDA to treat this debilitating, progressive and often disfiguring rare disease that typically begins early in life.

The FDA has granted approval of Koselugo to AstraZeneca Pharmaceuticals LP.

Neurofibromatosis type 1 is a condition characterized by changes in skin coloring (pigmentation) and the growth of tumors along nerves in the skin, brain, and other parts of the body. The signs and symptoms of this condition vary widely among affected people.

Beginning in early childhood, almost all people with neurofibromatosis type 1 have multiple café-au-lait spots, which are flat patches on the skin that are darker than the surrounding area.

"Everyone's daily lives have been disrupted during the COVID-19 pandemic, and in this critical time we want patients to know that the FDA remains committed to making patients with rare tumors and life threatening diseases, and their unique needs, a top priority. We continue to expedite product development for these patients," said Richard Pazdur, M.D., director of the FDA's Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA's Center for Drug Evaluation and Research.

Koselugo is approved specifically for patients who have symptomatic, inoperable plexiform neurofibromas (PN), which are tumors involving the nerve sheaths (coating around nerve fibers) and can grow anywhere in the body, including the face, extremities, areas around the spine and deep in the body where they may affect organs. Koselugo is a kinase inhibitor, meaning it functions by blocking a key enzyme, which results in helping to stop the tumor cells from growing.

NF1 is a rare, progressive condition caused by a mutation or flaw in a particular gene. NF1 is usually diagnosed in early childhood and appears in an estimated 1 out of every 3,000 infants. It is characterized by changes in skin coloring (pigmentation), neurologic and skeletal impairments and risk for development of benign and malignant tumors throughout life. Between 30% and 50% of patients born with NF1 develop one or more PNs.

"We are committed to regulatory flexibility and providing extensive guidance to industry in an effort to bring drugs forward that fulfill unmet medical needs. Koselugo represents this commitment," noted Pazdur. "For the first time, pediatric patients now have an FDA-approved drug to treat plexiform neurofibroma, a rare tumor associated with NF1."

The FDA approved Koselugo based on a clinical trial conducted by the National Cancer Institute of pediatric patients who had NF1 and inoperable PN (defined as a PN that could not be completely removed without risk for substantial morbidity to the patient). The efficacy results were from 50 of the patients who received the recommended dose and had routine evaluations of changes in tumor size and tumor-related morbidities during the trial. Patients received Koselugo 25 mg/m2 orally twice a day until disease progression or until they experienced unacceptable adverse reactions. The clinical trial measured the overall response rate (ORR), defined as the percentage of patients with a complete response and those who experienced more than a 20% reduction in PN volume on MRI that was confirmed on a subsequent MRI within 3-6 months. The ORR was 66% and all patients had a partial response, meaning that no patients had complete disappearance of the tumor. Of these patients, 82% had a response lasting 12 months or longer.

Other clinical outcomes for patients during Koselugo treatment including changes in PN-related disfigurement, symptoms and functional impairments. Although the sample sizes of patients assessed for each PN-related morbidity (such as disfigurement, pain, strength and mobility problems, airway compression, visual impairment and bladder or bowel dysfunction) were small, there appeared to be a trend of improvement in PN-related symptoms or functional deficits during treatment.

Common side effects for patients taking Koselugo were vomiting, rash, abdominal pain, diarrhea, nausea, dry skin, fatigue, musculoskeletal pain (pain in the body affecting bones, muscles, ligaments, tendons and nerves), fever, acneiform rash (acne), stomatitis (inflammation of the mouth and lips), headache, paronychia (infection in the skin that surrounds a toenail or fingernail) and pruritus (itching).

Koselugo can also cause serious side effects including heart failure (manifested as ejection fraction decrease, or when the muscle of the left ventricle of the heart is not pumping as well as normal) and ocular toxicity (acute and chronic damage to the eye) including retinal vein occlusion, retinal pigment epithelial detachment and impaired vision. Patients should have cardiac and ophthalmic assessments performed prior to initiating Koselugo and at regular intervals during treatment. Koselugo can also cause increased creatinine phosphokinase (CPK). CPK is an enzyme found in the heart, brain and skeletal muscles. When muscle tissue is damaged, CPK leaks into a person's blood. CPK elevation in a patient receiving Koselugo should prompt an evaluation for rhabdomyolysis (breakdown of skeletal muscle due to direct or indirect muscle injury). Koselugo should be withheld, dosage reduced or dosage permanently discontinued based on the severity of adverse reactions. Further, Koselugo contains Vitamin E, and patients are at an increased risk of bleeding if their daily intake of Vitamin E exceeds the recommended or safe limits.

Based on findings from animal studies, Koselugo may cause harm to a newborn baby when administered to a pregnant woman. The FDA advises health care professionals to tell females of reproductive age, and males with female partners of reproductive potential, to use effective contraception during treatment with Koselugo, and for one week after the last dose.

The FDA granted this application Priority Review and Breakthrough Therapy designation. Koselugo also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases, and Rare Pediatric Disease Designation for the treatment of pediatric NF1. The application is awarded a Rare Pediatric Disease Priority Review Voucher.

Neurofibromatosis children Food and drug administration US food and drug administration #Selumetinib #Koselugo 
Source : FDA
Dr. Kamal Kant Kohli
Dr. Kamal Kant Kohli

    Dr Kamal Kant Kohli-MBBS, DTCD- a chest specialist with more than 30 years of practice and a flair for writing clinical articles, Dr Kamal Kant Kohli joined Medical Dialogues as an Editor-in-Chief for the Speciality Medical Dialogues section. Besides writing articles, as an editor, he proofreads and verifies all the medical content published on Medical Dialogues including those coming from journals, studies,medical conferences,guidelines etc. Before Joining Medical Dialogues, he has served at important positions in the medical industry in India including as the Hony. Secretary of the Delhi Medical Association as well as the chairman of Anti-Quackery Committee in Delhi and worked with other Medical Councils in India. Email: editorial@medicaldialogues.in. Contact no. 011-43720751

    Show Full Article
    Next Story
    Similar Posts
    NO DATA FOUND

    Editorial

    Treatment challenges in managing Gastroparesis related to PPI Use: Analyzing scope of Probiotics

    Treatment challenges in managing Gastroparesis related to PPI Use: Analyzing scope of Probiotics

    Review: Efficacy of Antiviral Agents Against  Omicron Subvariant BA.2

    Review: Efficacy of Antiviral Agents Against Omicron Subvariant BA.2

    Choice of Antibiotics in Common Infections: Doxycycline or Azithromycin?

    Choice of Antibiotics in Common Infections: Doxycycline or Azithromycin?

    Role of acyclovir in Management of chickenpox: Analyzing the importance of starting early

    Role of acyclovir in Management of chickenpox: Analyzing the importance of starting early

    Parkinsonism and the place of Amantadine - Review Analysis

    Parkinsonism and the place of Amantadine - Review Analysis

    View All

    Journal Club Today

    Health News Today

    Health Bulletin 17/May/2022

    Health Bulletin 17/May/2022

    View All
    © 2019 All Rights Reserved.
    Powered By: Hocalwire
    We use cookies for analytics, advertising and to improve our site. You agree to our use of cookies by continuing to use our site. To know more, see our Cookie Policy and Cookie Settings.Ok
    X
    X