Medical Dialogues
  • Dermatology
Login Register
This site is intended for healthcare professionals only
Login Register
  • Medical Jobs
  • Medical Matrimony
  • MD Brand Connect
  • 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
    • Medico Legal Update
    • Nephrology
    • Neurology and Neurosurgery
    • Obstetrics and Gynaecology
    • Oncology
    • Ophthalmology
    • Orthopaedics
    • Pediatrics & Neonatology
    • Psychiatry
    • Pulmonology
    • Radiology
    • Surgery
    • Top Videos
    • Urology
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
    • Pharmacy Education 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
      • Medico Legal Update
      • 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
LoginRegister
Medical Dialogues
LoginRegister
  • 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
    • Pharmacy Education News
  • Case of the Day
  • Editorial
  • Home
  • Pharmaceuticals
  • FDA rejects BioMarin's ...

FDA rejects BioMarin's muscle wasting drug; Sarepta drug in focus

Written by savita thakur thakur Published On 2016-01-15T09:15:50+05:30  |  Updated On 15 Jan 2016 3:45 AM GMT
FDA rejects BioMarins muscle wasting drug; Sarepta drug in focus
  • Facebook
  • Twitter
  • Whatsapp
  • Telegram
  • Linkedin
  • Email
  • koo
  • Facebook
  • Twitter
  • Whatsapp
  • Telegram
  • Linkedin
  • Email
  • koo
  • Facebook
  • Twitter
  • Whatsapp
  • Telegram
  • Linkedin
  • Email
  • koo

The U.S. Food and Drug Administration rejected BioMarin Pharmaceutical Inc's drug to treat a rare muscle-wasting disorder, shifting investor focus to Sarepta Therapeutics Inc's rival drug in development.The rejection caps months of uncertainty surrounding the drug, Kyndrisa, which was billed to be the first treatment in the United States for Duchenne muscular dystrophy (DMD).DMD is a...

The U.S. Food and Drug Administration rejected BioMarin Pharmaceutical Inc's drug to treat a rare muscle-wasting disorder, shifting investor focus to Sarepta Therapeutics Inc's rival drug in development.

The rejection caps months of uncertainty surrounding the drug, Kyndrisa, which was billed to be the first treatment in the United States for Duchenne muscular dystrophy (DMD).

DMD is a progressive, degenerative genetic disorder that hampers muscle movement and affects one in 3,600 newborn boys. Most patients die by the age of 30. DMD has no cure.

A panel of independent advisers to the FDA had in November indicated that Kyndrisa's effectiveness in trials was not enough to warrant approval. Sarepta's drug, eteplirsen, is up for review next week.

WBB Securities analyst Steve Brozak said Kyndrisa's rejection effectively crowned Sarepta the U.S. DMD winner and that pressure to approve a treatment would mount.

Analysts said BioMarin will likely need another late-stage study for Kyndrisa in the United States. Kyndrisa is also under review in Europe.

Sarepta's eteplirsen, like BioMarin's Kyndrisa, skips a faulty section of the gene to produce dystrophin, the lack of which is believed to cause DMD.

Both drugs target a particular subset of DMD patients, which translates into an addressable population of about 1,800 boys in the United States and about 5,000 outside, according to Wedbush Securities analyst Heather Behanna.

The U.S. rejection, however, is not end of the road for Kyndrisa. BioMarin expects to hear back from the European regulatory advisory committee in April or May, with a final decision in the third quarter.

While a U.S. approval would have given BioMarin first-mover advantage in the market, a European approval is perhaps more significant as the drug will target a larger patient population.

Evercore ISI's Mark Schoenebaum expects BioMarin make its next move only after the FDA's decision on Sarepta, and European regulators' decision on Kyndrisa.

BioMarin said on Thursday it would work with the FDA to determine its next step.

PTC Therapeutics Inc's DMD drug, Translarna, won conditional European approval in 2014, to treat a different subset of patients.

Last year, PTC said it had enough data to submit a U.S. marketing application, despite the drug failing a U.S. late-stage study.
BioMarin BioMarin Pharmaceutical muscle wasting drug Sarepta Therapeutics Inc USFDA 
savita thakur thakur
savita thakur thakur
    Show Full Article
    Next Story
    Similar Posts
    NO DATA FOUND

    Editorial

    Management of Allergic Rhinitis during COVID-19 Pandemic

    Management of Allergic Rhinitis during COVID-19 Pandemic

    Role of Probiotics in recurrent bacterial vaginosis: A review

    Role of Probiotics in recurrent bacterial vaginosis: A review

    Bronchial Asthma - Early Indicators for Diagnosis and Its Management in Indian Practice Settings

    Bronchial Asthma - Early Indicators for Diagnosis and Its Management in Indian Practice Settings

    Role of metoprolol tartrate in management of acute myocardial infarction in the present era

    Role of metoprolol tartrate in management of acute myocardial infarction in the present era

    Managing co-existing allergic rhinitis and asthma: Analyzing the role of Montelukast-levocetirizine combination

    Managing co-existing allergic rhinitis and asthma: Analyzing the role of Montelukast-levocetirizine...

    View All

    Journal Club Today

    Spending time online to help boosting childrens well-being

    Spending time online to help boosting children's well-being

    View All

    Health News Today

    Health Bulletin 27/June/2022

    Health Bulletin 27/June/2022

    View All
    © 2022 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