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What Is The Good News for NPS Pharmaceuticals’ Orphan Drug Gattex (Teduglutide) For Short Bowel Syndrome ?

December 21, 2012

       For the past 6 months, the Orphan Druganaut Blog has followed the journey of NPS Pharmaceuticals’ orphan drug Gattex (Teduglutide)/Revestive  for Short Bowel Syndrome (SBS), from FDA Orphan Drug Designation (ODD) to approval.      

       Per a December 21, 2012 Press Release, NPS Pharmaceuticals announces that the FDA approves Gattex (Teduglutide [rDNA Origin]) for injection, for subcutaneous use for the treatment of Short Bowel Syndrome (SBS), for adults who are dependent on parenteral support . This approval comes a week early, as the FDA PDUFA date was scheduled for on or before next Friday, December 28, 2012.             

        Patients who use Gattex, have a potential increased risk of developing cancer and abnormal growths (polyps) and obstructions in the intestine, gallbladder disease, and other side effects. Thus, Gattex is approved with a ”Risk Evaluation and Mitigation Strategy, consisting of a communication plan and training for prescribers”.  

        The FDA is requiring a postmarket study of SBS patients treated with the drug for the purpose of studying Gattex’ s long-term safety profile. Patients in this study will be followed for at least 10 years.

       Gattex, a first-in-class therapy, is the 1st major long-term treatment advance for SBS in nearly 40 years. Gattex is to be available in the 1st Quarter of 2013. It is the 3rd FDA-approved drug to treat adults with SBS receiving nutritional support. Zorbtive (somatropin) and Nutrestore (glutamine) were approved in 2003 and 2004, respectively.

       Francois Nader, MD, President & CEO of NPS Pharmaceuticals, says that, “ …. The approval of Gattex is a crowning achievement for our company and the catalyst for our transformation into a premier orphan drug business …… We  have also launched NPS Advantage, our free support program that includes, care coordinators who will work closely with patients …”.

       At the September 12, 2012 Morgan Stanley Global healthcare Conference, Mr. Nader discusses the steps for the commercialization of Gattex :

1. To find acute patients by reaching out to them one at a time

2. Use of social media to reach out to patients & advocacy groups

3. Engagement of the payers

4. Offering of internal help & “personalized service to patients” – “care coordinators” – to provide patients with support for how to navigate reimbursement process, how to use Gattex, how to reduce PN, etc.

5. A very narrow specialized targeted distribution with multiple players (GI physicians, home healthcare person, pharmacy, etc.).

      The FDA Regulatory History of Gattex :

  1. FDA Orphan Drug Designation (ODD) on June 29, 2000
  2. Original PDUFA date is September 30, 2012, but is extended by 3 months to December 30, 2012, to give reviewers additional time
  3. On October 16, 2012, the FDA GI Advisory Committee unanimously recommends Gattex for approval for SBS
  4. FDA approves Gattex on December 21, 2012.

         The  European Medicines Agency (EMA) Regulatory History for orphan drug Revestive (Teduglutide) :

  1. The positive opinion EMA’s Committee for Medicinal Products for Human Use (CHMP) issues on June 21, 2012 for Revestive for adults with SBS
  2. Takeda Pharmaceuticals and NPS Pharmaceuticals jointly announces on September 4, 2012 in a Press Release, that the European Commission (EC) grants European market authorization for teduglutide (Revestive in Europe; Gattex in US) as a once-daily treatment for adults with Short Bowel Syndrome (SBS)
  3. EMA issues EPAR on September 24, 2012.

Next Catalyst Event

  1. NPS Pharmaceuticals to host conference call on January 2, 2013.

References

Orphan Druganaut October 25, 2012 Blog Post titled, “Jim Cramer Interviews NPS Pharmaceuticals CEO On Orphan Drugs: CNBC Video”

Orphan Druganaut October 22, 2012 Blog Post titled, “NPS Pharmaceuticals’ Orphan Drug Gattex (Teduglutide) Study Results Presented At The American College of Gastroenterology (ACG) Meeing”

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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