FDA News, Week of Aug 24 and Aug 31, 2015 – Drug and Device Digest

Kicking off the PDUFA VI Reauthorization Process

FDA Voice

Highlights of progress:

  • FDA continues to meet or exceed review goals.
  • High rates of approvals for novel products during first submission – including rare diseases ( 17 orphan drug approvals in 2014).
  • Improved and increased communication between FDA and sponsors
  • implementation of a structured risk-benefit framework for reviews
  • Patient-Focused Drug Development program has been successful in systematically obtaining patient perspectives on certain diseases and related treatments.

Ideas for further enhancements:

  • Involve patient perspective in drug development processes;
  • Expand FDA’s Sentinel System for active surveillance of safety issues
  • Enhancing regulatory science initiatives, including the use of patient-reported outcomes and biomarkers.

SUMMARY : Tracking and Optimization of FDA Drug review will be critical components for the PDUFA VI re0authorizaiton


FDA approved

Promacta (Eltrombopag) : Extension of use in yound children with rare blood disorder  ( GlaxoSmithKline, Research Triangle Park, NC).

Designation: Orphan, Priority Review

Indication :  Treatment of thrombocytopenia in adult and pediatric patients 1 year and older with chronic immune (idiopathic) thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy

Description : Eltrombopag olamine, a small molecule thrombopoietin (TPO) receptor agonist, interacts with the transmembrane domain of the TPO receptor (also known as cMpl) leading to increased platelet production

Efficacy : Patients ages one to 17 years with chronic ITP

  • Two double-blind, placebo-controlled trials, n=159
  • More pediatric patients treated with PROMACTA (75%) compared with placebo (21%) had imporved platetelt counts (Prespecified)
  • Fewer treated patients required rescue treatment

Safety :

  • Liver Enzyme elevation : An elevation of ALT greater than or equal to 3x upper limit of normal occurred in 5% of patients in the eltrombopag group, and of those 2% had increases in ALT greater than or equal to 5x upper limit of normal.
  • No deaths or thromboembolic events

Varubi  (rolapitant) for nausea and vomiting from chemotherapy ( Tesaro Inc., Waltham, MA)

Designation : Standard

Indication: In combination with other antiemetic agents in adults for the prevention of delayed nausea and vomiting associated with initial and repeat courses of emetogenic cancer chemotherapy, including, but not limited to, highly emetogenic chemotherapy

Description: Substance P/neurokinin-1 (NK-1) receptor antagonist. Activation of NK-1 receptors plays a central role in nausea and vomiting induced by certain cancer chemotherapies, particularly in the delayed phase.


  • Three multicenter, randomized, double-blind, parallel group, controlled clinical studies, n=2800
  • Compare VARUBI regimen (VARUBI, granisetron and dexamethasone) vs Control (placebo, granisetron and dexamethasone)
  • Greater reduction in vomiting and use of rescue medicarion with varubi regimen


  • Contraindication : Varubi inhibits the CYP2D6 enzyme, Contraindicated with the use of thioridazine (metabolized bt CYP2D6); combination can cause abnormal heart rhythm that can be serious.
  • Other : Low white blood cell count (neutropenia), hiccups, decreased appetite, dizziness

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