Skip to main content
Subscribe
  • Sign Up Free
  • Login
  • Subscribe
  • News
    • Current News
    • Providers
    • Insurance
    • Government
    • Finance
    • Technology
    • Safety & Quality
    • Digital Health
    • Transformation
    • ESG
    • People
    • Regional News
    • Digital Edition (Web Version)
    • Patients
    • Operations
    • Care Delivery
    • Payment
    • Midwest
    • Northeast
    • South
    • West
  • Blogs
    • AI
    • Deals
    • Layoff Tracker
    • HIMSS 2023
  • Opinion
    • Breaking Bias
    • Commentaries
    • Letters
    • From the Editor
  • Events & Awards
    • Awards
    • Conferences
    • Galas
    • Virtual Briefings
    • Webinars
    • Nominate/Eligibility
    • 100 Most Influential People
    • 50 Most Influential Clinical Executives
    • Best Places to Work in Healthcare
    • Excellence in Governance
    • Health Care Hall of Fame
    • Healthcare Marketing Impact Awards
    • Top 25 Emerging Leaders
    • Top Innovators
    • Diversity in Healthcare
      • - Luminaries
      • - Top 25 Diversity Leaders
      • - Leaders to Watch
    • Women in Healthcare
      • - Luminaries
      • - Top 25 Women Leaders
      • - Women to Watch
    • Digital Health Transformation Summit
    • ESG: The Implementation Imperative Summit
    • Leadership Symposium
    • Social Determinants of Health Symposium
    • Women Leaders in Healthcare Conference
    • Best Places to Work Awards Gala
    • Health Care Hall of Fame Gala
    • Top 25 Diversity Leaders Gala
    • Top 25 Women Leaders Gala
    • - Hospital of the Future
    • - Value Based Care
    • - Hospital at Home
    • - Workplace of the Future
    • - AI and Digital Health
    • - Future of Staffing
    • - Hospital of the Future (Fall)
  • Multimedia
    • Podcast - Beyond the Byline
    • Sponsored Podcast - Healthcare Insider
    • Sponsored Video Series - One on One
    • Sponsored Video Series - Checking In with Dan Peres
  • Data & Insights
    • Data & Insights Home
    • Hospital Financials
    • Staffing & Compensation
    • Quality & Safety
    • Mergers & Acquisitions
    • Data Archive
    • Resource Guide: By the Numbers
    • Surveys
    • Data Points
  • Newsletters
  • MORE+
    • Contact Us
    • Advertise
    • Media Kit
    • Jobs
    • People on the Move
    • Reprints & Licensing
MENU
Breadcrumb
  1. Home
  2. Technology
May 12, 2015 12:00 AM

FDA panel recommends approving new cystic fibrosis treatment

Steven Ross Johnson
  • Tweet
  • Share
  • Share
  • Email
  • More
    Reprints Print

    A Food and Drug Administration advisory panel Tuesday recommended approving a new treatment for cystic fibrosis. Experts say the drug will reap billions for its manufacturer. The price of the medication will easily eclipse that of Gilead's $80,000 hepatitis C breakthrough drug, Sovaldi.

    The FDA's Pulmonary-Allergy Drugs Advisory Committee voted 12-1 to recommend approval of Vertex Pharmaceutical's drug Orkambi, a therapy that combines the already approved medication for cystic fibrosis, Kalydeco, with a newly developed medication called lumacaftor to treat the most common genetic mutation that causes cystic fibrosis.

    “Today's positive recommendation brings the cystic fibrosis community one step closer to potential approval of the first medicine to treat the underlying cause of this disease for many more people," said Vertex's executive vice president and chief medical officer, Dr. Jeffrey Chodakewitz, in a released statement.

    The drug would treat an estimated 8,500 of the 30,000 patients with CF in the U.S., according to documents released prior to the meeting (PDF). Estimated to affect 70,000 patients worldwide, cystic fibrosis is a life-threatening genetic condition that causes cells to secrete bodily fluids that become thick, blocking passageways in the lungs and pancreas.

    Kalydeco currently treats an estimated 2,000 patients who have a rarer form of the disease than the one Orkambi would treat.

    Conclusions from the committee's report on Orkambi suggest there are questions regarding one of the medication's benefits toward improving lung function. A clinical trial involving more than 1,000 patients showed the two-drug combination therapy resulted in modest improvement of lung function, with investigators stating, “The contribution of lumacaftor to the efficacy of the proposed combination products has not been shown.”

    Approval of Orkambi is vital for Cambridge, Mass.-based Vertex, which has posted a net loss of more than $198 million for the first quarter of 2015. Sales of Kalydeco, currently its only drug on the market, reached $130 million during the first quarter, ended March 31.

    “With this launch, they'll get to profitability almost instantaneously,” said Katherine Xu, a partner with the equity research firm William Blair & Co. who analyzes Vertex. “It's a tremendous opportunity for them.”

    Analysts have estimated sales of Orkambi could reach as high as $8 billion worldwide, though questions remain as to what price the company will place on the drug if it gets approved. Many feel the cost of the treatment will match the price of Kalydeco, which costs around $300,000 for a year's worth of treatment.

    Xu says the price Vertex ultimately sets for Orkambi will be determined by a number of factors that set it apart from its other medication, Kalydeco, an orphan drug whose price is high, in part, due to the small population the medication treats. Xu has estimated the initial price for Orkambi to be in the range of $200,000 to $300,000 per patient annually. A larger pool of potential patients usually helps bring down the cost of a drug, but the fact that the drug is a combination of two compounds, one of which is already being sold, may cause the initial price to be higher.

    “You're adding a drug to the existing drug in this case,” Xu said. “So, are you going to add a drug and then set a lower price? We just haven't seen that kind of precedent before.”

    The FDA is scheduled to issue its decision on Orkambi's approval on July 5. In such cases, the agency tends to follow the panel's recommendation, but it has been known to occasionally deviate from the position of its advisory body.

    Letter
    to the
    Editor

    Send us a letter

    Have an opinion about this story? Click here to submit a Letter to the Editor, and we may publish it in print.

    Recommended for You
    doctor_patient_tablet_getty_i_i.jpg
    How AI may influence pain medication prescriptions
    Amir Dan Rubin
    Amazon's One Medical CEO to step down later this year
    Most Popular
    1
    CMS tries luring providers to revamped Medicare ACOs
    2
    Oregon joins other states in setting ratios for nurse staffing
    3
    Blue Shield CA taps Amazon, Mark Cuban, CVS for new PBM model
    4
    A health innovation hub grows in Lake Nona Medical City
    5
    Hospital-at-home providers push for Medicaid coverage
    Sponsored Content
    Digital Health Intelligence Newsletter: Sign up to receive a twice-weekly (T, F) morning newsletter featuring the latest reporting on technologies, trends, players and money fueling the rapid changes in how healthcare is developed, paid for and delivered.
    Get Newsletters

    Sign up for enewsletters and alerts to receive breaking news and in-depth coverage of healthcare events and trends, as they happen, right to your inbox.

    Subscribe Today
    MH Magazine Cover

    MH magazine offers content that sheds light on healthcare leaders’ complex choices and touch points—from strategy, governance, leadership development and finance to operations, clinical care, and marketing.

    Subscribe
    Connect with Us
    • LinkedIn
    • Twitter
    • Facebook
    • RSS

    Our Mission

    Modern Healthcare empowers industry leaders to succeed by providing unbiased reporting of the news, insights, analysis and data.

    Contact Us

    (877) 812-1581

    Email us

     

    Resources
    • Contact Us
    • Help Center
    • Advertise with Us
    • Ad Choices
    • Sitemap
    Editorial Dept
    • Submission Guidelines
    • Code of Ethics
    • Awards
    • About Us
    Legal
    • Terms and Conditions
    • Privacy Policy
    • Privacy Request
    Modern Healthcare
    Copyright © 1996-2023. Crain Communications, Inc. All Rights Reserved.
    • News
      • Current News
      • Providers
      • Insurance
      • Government
      • Finance
      • Technology
      • Safety & Quality
      • Digital Health
      • Transformation
        • Patients
        • Operations
        • Care Delivery
        • Payment
      • ESG
      • People
      • Regional News
        • Midwest
        • Northeast
        • South
        • West
      • Digital Edition (Web Version)
    • Blogs
      • AI
      • Deals
      • Layoff Tracker
      • HIMSS 2023
    • Opinion
      • Breaking Bias
      • Commentaries
      • Letters
      • From the Editor
    • Events & Awards
      • Awards
        • Nominate/Eligibility
        • 100 Most Influential People
        • 50 Most Influential Clinical Executives
        • Best Places to Work in Healthcare
        • Excellence in Governance
        • Health Care Hall of Fame
        • Healthcare Marketing Impact Awards
        • Top 25 Emerging Leaders
        • Top Innovators
        • Diversity in Healthcare
          • - Luminaries
          • - Top 25 Diversity Leaders
          • - Leaders to Watch
        • Women in Healthcare
          • - Luminaries
          • - Top 25 Women Leaders
          • - Women to Watch
      • Conferences
        • Digital Health Transformation Summit
        • ESG: The Implementation Imperative Summit
        • Leadership Symposium
        • Social Determinants of Health Symposium
        • Women Leaders in Healthcare Conference
      • Galas
        • Best Places to Work Awards Gala
        • Health Care Hall of Fame Gala
        • Top 25 Diversity Leaders Gala
        • Top 25 Women Leaders Gala
      • Virtual Briefings
        • - Hospital of the Future
        • - Value Based Care
        • - Hospital at Home
        • - Workplace of the Future
        • - AI and Digital Health
        • - Future of Staffing
        • - Hospital of the Future (Fall)
      • Webinars
    • Multimedia
      • Podcast - Beyond the Byline
      • Sponsored Podcast - Healthcare Insider
      • Sponsored Video Series - One on One
      • Sponsored Video Series - Checking In with Dan Peres
    • Data & Insights
      • Data & Insights Home
      • Hospital Financials
      • Staffing & Compensation
      • Quality & Safety
      • Mergers & Acquisitions
      • Data Archive
      • Resource Guide: By the Numbers
      • Surveys
      • Data Points
    • Newsletters
    • MORE+
      • Contact Us
      • Advertise
      • Media Kit
      • Jobs
      • People on the Move
      • Reprints & Licensing