Drug and Device Approvals: YESCARTA, IMPELLA RP – Drug and Device Digest

Week of October 16, 2017


YESCARTA™ (axicabtagene ciloleucel) suspension for intravenous infusion

Kite Pharma, Inc. (Gilead company)

INDICATION: Treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.

Limitation of Use: Not indicated for the treatment of patients with primary central nervous system lymphoma


  • DLBCL most common type of NHL in adults;  72,000 new cases of NHL diagnosed in the U.S. each year, and DLBCL represents approximately one in three cases
  • Milestone in the development of a whole new scientific paradigm for the treatment of serious diseases
  • Second gene therapy approved by the FDA; first for certain types of non-Hodgkin lymphoma (NHL)


  • Orphan Drug Designation; no pediatric requirements
  • Postmarketing requirements: Assessment of long-term safety
  • REMS requirements:  Elements to assure safe use, implementation system
    and timetable for submission of REMS assessments

MECHANISM OF ACTION: CD19-directed genetically modified autologous T cell immunotherapy, binds to CD19-expressing cancer cells and normal B cells. Studies demonstrated that following anti-CD19 CAR T cell engagement with CD19-expressing target cells leads to sequence of events killing of CD19-expressing cells

ADMINISTRATION: Each dose is a customized treatment created using a patient’s own immune system to help fight the lymphoma. The patient’s T-cells, a type of white blood cell, are collected and genetically modified to include a new gene that targets and kills the lymphoma cells. Once the cells are modified, they are infused back into the patient


  • Single-arm, open-label, multicenter trial, n=100 patients with relapsed or refractory aggressive B-cell non-Hodgkin lymphoma, single infusion of YESCARTA
  • Efficacy: Complete remission (CR) rate, duration of response (DOR)
  • CR= 52 patients, median DOR= 9.2 months


  • Boxed Warning: Cytokine release syndrome (CRS) and neurologic toxicities that can be fatal or life-threatening
  • Other side effects: Serious infections, low blood cell counts, weakened immune system. Side effects from treatment with Yescarta usually appear within the first one to two weeks
  • REMS : Certification of  hospitals and their associated clinics to recognize and manage CRS and nervous system toxicities


  • Available only in authorized treatment centers
  • Ongoing and active discussions with payers
  • Pricing based on discussions with government agencies that reimburse for drug costs and private insurers, and cancer centers.





INDICATION FOR USE: Providing temporary right ventricular support for up to 14 days in patients with a body surface area ≥1.5 m2, who develop acute right heart failure or decompensation following left ventricular assist device implantation, myocardial infarction, heart transplant, or open-heart surgery

ADDRESSING UNMET NEED: Smallest heart pump for right heart failure


  • Regulation Number: 870.4360
  • Product Code: PYX
  • Postmarketing requirement: Real-world evidence on safety and effectiveness

DESCRIPTION: Comprised of three components

  • Impella RP Catheter: a 22 Fr micro-axial flow pump catheter and its accessories
  • Automatic Impella Controller (AIC): a reusable external drive console
  • Impella Purge Cassette: an infusion pump used to flush the Impella RP Catheter


  • Pooled from the following three (3) datasets: Impella RP System pivotal study: 30 patients, Impella RP System continued access protocol (CAP) study: 4 patients,  Impella RP System post-approval study (PAS): 26 patients
  • Use of System to provide percutaneous hemodynamic support for right heart failure: Survival rate of 73.3% at 30 days
  • Main adverse events:  Major bleeding and hemolysis, no pulmonary embolism


  • Hospital ICD-10 procedure code specific to percutaneous heart assist
  • Separate physician payment for insertion, repositioning, and removal (CPT)
  • Coverage by Medicare and major payers


Image Credits: Kite, Abiomed



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