FDA Market Authorization: CRYSVITA, TAVALISSE, MALDI Biotyper, CARDIOFORM Septal Occluder, GUARDIAN Connect System  – Drug and Device Digest


CRYSVITA (burosumab-twza) injection

Ultragenyx Pharmaceutical, Inc.

INDICATION:  Treatment of X-linked hypophosphatemia (XLH) in adult and pediatric
patients 1 year of age and older.


  • XLH, a rare, inherited form of rickets, affects ~ 3,000 children and 12,000 adults in US
  • XLH differs from other forms of rickets in that vitamin D therapy is not effective
  • First drug approved to treat adults and children ages 1 year and older with XLH 

MECHANISM OF ACTION: Binds to and inhibits the biological activity of FGF23 restoring renal phosphate reabsorption and increasing the serum concentration of 1,25 dihydroxy vitamin D.


  • 65 pediatric patients and 134 adults with XLH in 4 studies, CRYSVITA vs, placebo,
  • Achievement of normal phosphorus levels: Adults: 94% adults vs. 8%, Children: 94-100%
  • Improved radiographic evaluation of rickets vs.  natural history cohort: In both children and adults,


  • Most common adverse reactions: Back pain, headache, restless leg syndrome, decreased vitamin D, dizziness and constipation
  • Most common adverse reactions in children: Headache, injection site reaction, vomiting, decreased vitamin D and pyrexia


  • Orphan designation, Breakthrough therapy designation
  • Pediatric Priority Review Voucher granted
  • Exempt from required pediatric assessments
  • Postmarketing requirements: Post-approval surveillance program with safety objectives, lactation sub-study in lactating women ,  reanalyze banked immunogenicity serum samples

REIMBURSEMENT PATHWAY: For rare disease indication

  • Covered with limited issue in Medicaid and Medicare
  • However, increased scrutiny of rare disease therapies and evolution of
    precision medicine; need to articulate long-term benefit of early
    diagnosis and treatment to patient and the health care system



TAVALISSE (fostamatinib disodium hexahydrate) tablets

Rigel Pharmaceuticals, Inc.

INDICATION: Treatment of thrombocytopenia in adult patients with chronic immune
thrombocytopenia (ITP) who have had an insufficient response to a previous treatment


  • Two identical, double-blind, placebo-controlled trials, n=150,  patients with persistent or chronic ITP who had an insufficient response to previous treatment, TAVALISSE vs. placebo
  • Endpoint: Stable platelet response (at least 50 x109/L on at least 4 of the 6 visits between Weeks 14 to 24)
  • Study 1: 18% (n=9) vs, 0% (p=0.03)
  • Study 2: 16% (n=8) vs. 4% (n=1), (p=0.26)
  • Study 3: Stable response in 23% (n=10)
  • Durable platelet responses seen


  • Most common adverse reactions: Diarrhea, hypertension, nausea, dizziness, alanine aminotransferase/aspartate aminotransferase (ALT/AST) increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia
  • Serious adverse drug reactions: Febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis


  • Orphan desugnation
  • Postmarketing commitmenets: Quality assessments



MALDI Biotyper CA System

Bruker Daltonik GmbH

INDICATION FOR USE: Mass spectrometer system using matrix-assisted laser
desorption/ionization – time of flight (MALDI-TOF) for the identification and differentiation of microorganisms cultured from human specimens.

The MALDI Biotyper CA System is a qualitative in vitro diagnostic device indicated for use in conjunction with other clinical and laboratory findings to aid in the diagnosis of bacterial and fungal infections – particularly  Candida auris (C. auris)


  • Emerging pathogen Candida auris (C. auris) can cause serious infections in hospitalized patients
  • Can cause serious infections in hospitalized patients (e.g., bloodstream infections) and is frequently resistant to multiple antifungal drugs used to treat Candida infections.

GENERIC DEVICE TYPE:  Clinical mass spectrometry microorganism identification and differentiation system

  • Qualitative in vitro diagnostic device intended for the identification and differentiation of microorganisms from processed human specimens. The system acquires, processes, and analyzes spectra to generate data specific to microorganism(s). The device is indicated for use in conjunction with other clinical and laboratory findings to aid in the diagnosis of bacterial and fungal infection.


  • Evaluated use of a standard protocol for adding C. auris to system database in conjunction with the performance data of 28 C. auris isolates (samples)
  •  C.auris isolates obtained from various culture collections, including the U.S. Centers for Disease Control and Prevention’s and FDA’s Antibiotic Resistance Isolate Bank.
  • System can reliably identify C. auris 100% of the time


  • Incorrect identification or lack of identification of a pathogenic microorganism: Special Controls
  • Failure to correctly interpret test results: Special Controls
  • Failure to correctly operate the instrument: Special Controls


  • Add to cleared uses for identification of 333 species or species groups, covering 424 clinically relevant bacteria and yeast species
  • Regulation Number: 21 CFR 866.3378
  • Regulation Name: Clinical Mass Spectrometry Microorganism Identification and Differentiation System
  • Regulatory Class: Class II
  • Product Code: QBN



GORE® CARDIOFORM Septal Occluder

W. L. Gore and Associates, Inc.

SUPPLEMENTAL INDICATION FOR USE: Permanently implanted device indicated for the percutaneous, transcatheter closure of the following defects of the atrial septum:

  • ostium secundum atrial septal defects (ASDs)
  • patent foramen ovale (PFO) to reduce the risk of recurrent ischemic stroke in patients, predominantly between the ages of 18 and 60 years, who have had a cryptogenic stroke due to a presumed paradoxical embolism, as determined by a neurologist and cardiologist following an evaluation to exclude known causes of ischemic stroke.


  • Implant (occluder) and delivery catheter (a small tube)
  • Occluder made of self-expanding, nickel-titanium (Nitinol) wires, covered in woven fabric
  • Occluder shaped as two discs connected in the cente
  • Occluder compressed to a small size to allow it to pass through the delivery catheter for implantation


  • Prospective, randomized (2:1), open-label, multi-center study, n=664, antiplatelet medical management and PFO closure with the GORE® CARDIOFORM Septal Occluder vs. antiplatelet medical management alone
  • Co-primary endpoints: Freedom from recurrent stroke and incidence of new brain infarction. PFO closure was associated with a statistically significant
    77% relative risk reduction in recurrent stroke
  • PFO closure was also associated with  a statistically significant 49% relative risk reduction in incidence of new brain infarction


  • No significant difference in overall rate of SAEs between the control (medical management) and device groups
  • Low rate of device- or procedure-related SAEs (3.6%)
  • Subjects had a higher incidence of atrial fibrillation or flutter (6.6%), but were non-serious
  • No device- or procedure related deaths.


  • For expanding the indications to include closure of the patent foramen
    ovale (PFO) to reduce the risk of recurrent ischemic stroke
  • Product Code: MLV
  • Postapproval studies: Safety through 5 years post-procedure, acute, subacute, and longterm safety and effectiveness


  • Approved CMS IDE study
  • Partnership with CODING STRATEGIES (an independent industry-leading resource in coding, coverage, and reimbursement education)



GUARDIAN Connect System 

Medtronic MiniMed, Inc.

INDICATION FOR USE: For continuous or periodic monitoring of glucose levels in the interstitial fluid under the skin, in patients (14 to 75 years of age) with diabetes mellitus.

Provides real-time glucose values and trends through a Guardian Connect app installed on a compatible consumer electronic mobile device. It allows users to detect trends and track patterns in glucose concentrations. The Guardian Connect app alerts if a Guardian Sensor (3) glucose level reaches, falls below, rises above, or is predicted to surpass set values.

The Guardian Sensor (3) glucose values are not intended to be used directly for making
therapy adjustments, but rather to provide an indication of when a finger stick may be
required. All therapy adjustments should be based on measurements obtained using a
home glucose monitor and not on values provided by the Guardian Sensor (3).


  • Provides real-time glucose values and trends through a Guardian Connect app installed on a compatible mobile device platform (e.g., iPhone or iPad)
  • Guardian Connect app is a mobile medical application that allows users to track patterns in glucose concentrations and to possibly identify episodes of low and high glucose
  • System is designed to provide continuous glucose monitoring for up to seven days
  • System consists primarily of a sensor, transmitter, and mobile medical app


  • Evaluate the performance of the Guardian Sensor (3) to support 7 days of use
  • Missed alerts and false negative hypoglycemia and hyperglycemic readings related to patients not being alerted to the need to perform a fingerstick to detect hypoglycemia or hyperglycemia


  • Device Procode: MDS
  • The Guardian Sensor (3) used with the Guardian Connect system is the same as the
    Guardian Sensor (3) used with the MiniMed 670G System (P160017) and the iniMed
    630G System (P150001/S008), which were previously approved


  • Continuous Glucose Monitors are reimbursed by CMS




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