Prescribe Right
Prescribe Right
  • Home
  • About
    • Endorsements
    • Published Articles
    • Consulting
    • Effective Pharmacotherapy
  • Pipeline News
    • Podcast
    • Recent Tweets
  • Pharmaceutical Pipeline Tracker
    • Access Pharmaceutical Pipeline Tracker
    • Pharmaceutical Pipeline Tracker Demo
    • Sample Drug Review
    • Case Studies >
      • Monitoring Drugs With PDUFA Dates
      • Monitoring Drugs By Therapeutic Category
      • Monitoring by Indication
      • Research a Single Drug
    • Sample Searches >
      • Single Drug Report Sample
      • Indication Search Sample
      • Drug Class Search Sample
      • Therapeutic Area Search Sample
      • Company Search Sample
  • Contact Us
  • Home
  • About
    • Endorsements
    • Published Articles
    • Consulting
    • Effective Pharmacotherapy
  • Pipeline News
    • Podcast
    • Recent Tweets
  • Pharmaceutical Pipeline Tracker
    • Access Pharmaceutical Pipeline Tracker
    • Pharmaceutical Pipeline Tracker Demo
    • Sample Drug Review
    • Case Studies >
      • Monitoring Drugs With PDUFA Dates
      • Monitoring Drugs By Therapeutic Category
      • Monitoring by Indication
      • Research a Single Drug
    • Sample Searches >
      • Single Drug Report Sample
      • Indication Search Sample
      • Drug Class Search Sample
      • Therapeutic Area Search Sample
      • Company Search Sample
  • Contact Us
Pipeline News and Updates
Picture

Drug to Treat Hereditary Transthyretin Amyloidosis

5/12/2018

 
Hereditary transthyretin amyloidosis (hATTR) is an inherited disorder that results in the gradual progressive deposit of abnormal protein called amyloid in the body's organs and tissues. Currently the only treatment for hATRR is liver transplant. Replacing the liver stops the production of abnormal transthyretin (TTR) proteins. Diflunisal is tired in some patients, but use is based on a single 130 patient trial. 
 
Three drugs in development, tafamidis, inotersen and patisiran, represent potential breakthroughs in the treatment of hATTR. Because no treatment outside of liver transplant exists, these three drugs may become preferred treatment choices. Patisiran shows some evidence of disease reversal, but all three drugs appear to have their greatest effect when started early. 
 
Tafamidis is a once daily oral drug, while inotersen is given as a weekly subcutaneous injection and patisiran is an intravenous infusion given every three weeks. All three drugs are investigational in the U.S., but tafamidis has been available in Europe since 2011. The FDA asked for additional clinical trials before the drug could be approved.
 
The CEO of Alnylam said that patisiran will have a six-figure price. Some analysts have projected a cost in the range of $200,000 to $400,000. Cost estimates for inotersen or tafamadis are not available. Analysts estimate that tafamidis may have a cost advantage over inotersen and patisiran.
 
In early May 2018, the FDA delayed review of inotersen to allow more time to evaluate the drug’s application. The drug will now be reviewed in the fall. The PDUFA date for patisiran is August 11. The NDA for tafamidis has not been filed. None of the pivotal trials for inotersen or patisiran have been published, so our first indication of the strength of the data may be when the FDA releases a review of the drugs.
 
The clinical effects of inotersen and patisiran are based on the elimination of all TTR production long-term toxicities. TTR transports thyroxine and retinol, so it will be important to monitor the results of long term trials to determine if a lack of TTR creates adverse events. 
 
These drugs act on TTR and not on correcting the mutation that causes the abnormal TTR to be produced.  Because the alternate is liver transplantation, the drugs will likely be the preferred treatment until a genome edit is created to correct the abnormal protein mutation.
    Stay informed, subscribe to the 
    ​
    Prescribe Right Pharmaceutical Pipeline Tracker
    Latest Tweets from Prescribe Right

    Archives

    March 2023
    February 2023
    January 2023
    December 2022
    October 2022
    September 2022
    August 2022
    July 2022
    June 2022
    May 2022
    April 2022
    March 2022
    February 2022
    January 2022
    December 2021
    November 2021
    October 2021
    September 2021
    August 2021
    July 2021
    June 2021
    May 2021
    April 2021
    March 2021
    February 2021
    January 2021
    December 2020
    November 2020
    October 2020
    September 2020
    August 2020
    July 2020
    June 2020
    May 2020
    April 2020
    March 2020
    February 2020
    January 2020
    December 2019
    November 2019
    October 2019
    September 2019
    August 2019
    July 2019
    June 2019
    May 2019
    April 2019
    March 2019
    February 2019
    January 2019
    December 2018
    November 2018
    October 2018
    September 2018
    August 2018
    June 2018
    May 2018
    April 2018
    March 2018
    February 2018
    January 2018
    December 2017
    November 2017
    October 2017
    September 2017
    August 2017
    August 2015

    RSS Feed

Services

Pharmaceutical Pipeline Tracker​
Consulting

Company

About
Blog
Tweets

Support

Contact
© COPYRIGHT 2015. ALL RIGHTS RESERVED.