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BRAINSWAY Deep Transcranial Magnetic Stimulation System

Brainsway Ltd.

INDICATION FOR USE: Adjunct for the treatment of adult patients suffering from Obsessive-Compulsive Disorder

ADDRESSING UNMET NEED: 

  • Transcranial magnetic stimulation has potential to help patients suffering from depression and headaches
  • Another option for patients with OCD who have not responded to traditional treatments

GENERIC DEVICE TYPE: Transcranial magnetic stimulation system for neurological and psychiatric disorders and conditions

  • Prescription, non-implantable device that uses brief duration, rapidly alternating,
    or pulsed, magnetic fields to induce neural activity in the cerebral cortex. It is not intended for applying or focusing magnetic fields towards brain areas outside cerebral cortex (e.g., cerebellum). A repetitive transcranial magnetic stimulation system that is intended to treat major depressive disorder is classified in § 882.5805. A transcranial magnetic stimulation system for headache is classified in § 882.5808.

EFFECTIVENESS & SAFETY: 

  • Multi-center study, n=100 patients receiving OCD treatments (medical management), Brainsway device vs. non-working (sham) device
  • Effectiveness endpoint: Reduction in Yale-Brown Obsessive Compulsive Scale (YBOCS) score; 38% on Brainsway with > 30% reduction vs. 11%
  • Adverse reactions: Headache,  application site pain or discomfort, jaw pain, facial pain, muscle pain, spasm or twitching, and neck pain

IDENTIFIED RISKS & MITIGATION MEASURES:

  • Seizure, Thermal injury, Hearing loss, Scalp discomfort, dizziness, nausea, pain in neck or jaw, headache, or other adverse effects due to treatment, Adverse tissue reaction, Electrical shock,  Device failure due to interference with other devices
  • Non-clinical performance testing, Labeling, Thermal safety testing, Electrical safety testing, Electromagnetic compatibility testing, Software verification, validation, and hazard analysis, Biocompatibility evaluation

REGULATORY PATHWAY: De Novo request

  • Prior approvals in treatment for major depression (2008) and treating pain associated with certain migraine headaches (2013)
  • Regulation Number: 21 CFR 882.5802
  • Regulation Name: Transcranial magnetic stimulation system for neurological and psychiatric disorders and conditions
  • Regulatory Class: Class II
  • Product Code: QCI

CLASSIFICATION ORDER


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Abbott RealTime IDH1

Abbott Molecular Inc.

INDICATION FOR USE:  In vitro polymerase chain reaction (PCR) assay for the qualitative detection of single nucleotide variants (SNVs) coding five IDH1 R132 mutations (R132C, R132H, R132G, R132S, and R132L) in DNA extracted from human blood (EDTA) or bone marrow (EDTA). Abbott RealTime IDH1 is for use with the Abbott m2000rt System.

Abbott RealTime IDH1 is indicated as an aid in identifying acute myeloid leukemia (AML) patients with an isocitrate dehydrogenase-1 (IDH1) mutation for treatment with TIBSOVO® (ivosidenib).

This test is for prescription use only.

COMPONENTS:  

  • Abbott RealTime IDH1 Amplification Reagent Kit
  • Abbott RealTime IDH1 Control Kit
  • Abbott mSample Preparation SystemDNA Kit
  • . Abbott RealTime IDH1 m2000rt Application CD-ROM

EFFECTIVENESS:

  • Accurate qualitative detection of single nucleotide variants (SNVs) coding five IDH1 mutations (R132C, R132H, R132G, R132S and R132L) in DNA extracted from human bone marrow or blood in patients with relapsed or refractory AML
  • Open-label, single-arm, international, multicenter clinical trial of TIBSOVO (ivosidenib), n=174 adult patients with relapsed or refractory AML and one of 5 IDH1 mutations in codon R132 detected by Abbott RealTime IDH1 assay
  • 32.8% exhibited complete remission or complete remission with partial hematological recovery

SAFETY:

  • Potential mismanagement of patients resulting from false results
  • Failure to perform as expected or failure to correctly interpret test results
  • False positive test result may lead to TIBSOVO treatment in patient who is not expected to benefit, and suffer from any potential adverse side effects
  • False negative test result may lead to TIBSOVO (ivosidenib) treatment not being administered to a patient who may benefit from this drug

REGULATORY PATHWAY: PMA, Device Procode: OWD

LABEL


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TAKHZYRO (lanadelumab-flyo) injection, for subcutaneous use

Dyax Corporation

INDICATION:  For prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients 12 years and older.

ADDRESSING UNMET NEED:

  • First monoclonal antibody approved to treat patients 12 years and older with types I and II HAE
  • HAE is rare and serious genetic disease that affects people with low levels of and poorly functioning C1-INH proteins; recurrent, unpredictable episodes of severe swelling in different areas of the body

MECHANISM OF ACTION:  Fully human monoclonal antibody (IgG1/κ-light chain) that binds plasma kallikrein (uncontrollably increased in HAE) and inhibits proteolytic activity which prevents angioedema attacks

EFFICACY:

  • Multicenter, randomized, double-blind, placebo-controlled, parallel-group study, n=125 patients with HAE
  • Takhzyro caused clinically meaningful and statistically significant reductions in the rate of investigator-confirmed HAE attacks vs. placebo over 6-month treatment period

SAFETY: 

  • Most common adverse drug reactions: Injection site reactions, upper respiratory infections, headache, rash, muscle pain, dizziness and diarrhea.

REGULATORY PATHWAY: BLA

  • Priority Review, Breakthrough Therapy designation, Orphan Drug designation
  • Postmarketing commitment: Low endotoxin recovery (LER) study

LABEL


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OXERVATE (cenegermin-bkbj) ophthalmic solution for topical
ophthalmic use 

Dompé farmaceutici SpA.

INDICATION: Treatment of neurotrophic keratitis

ADDRESSING UNMET NEED:

  • First approved drug for neurotrophic keratitis, a rare degenerative disease affecting cornea
  • Loss of corneal sensation impairs corneal health and damage- corneal thinning, ulceration, and perforation in severe cases
  • Prevalence estimated to be less than five in 10,000 individuals

MECHANISM OF ACTION: Nerve growth factor is an endogenous protein involved in differentiation and maintenance of neurons which acts through nerve growth factor receptors in the anterior segment of the eye to support corneal innervation and integrity

EFFICACY:

  • Two, eight-week, randomized controlled multi-center, double-masked studies. n= 151 patients with neurotrophic keratitis
  • Across both studies, complete corneal healing in eight weeks was demonstrated in 70% of patients treated with Oxervate (containing cenegermin) vs. 28% treated without cenegermin

SAFETY:

  • Most common adverse reactions: Eye pain, ocular hyperemia, eye inflammation and increased lacrimation (wat

REGULATORY PATHWAY: BLA

  • Priority Review, Orphan Drug Designation
  • Postmarketing commitments:  Clinical study to determine systemic exposure following repeated topical ocular dosing, manufacturing and shipping validations

LABEL


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DIACOMIT (stiripentol) capsules and powder, for oral suspension

Biocodex

INDICATION: Treatment of seizures associated with Dravet syndrome (DS) in patients 2 years of age and older taking clobazam.

There are no clinical data to support the use of DIACOMIT as monotherapy in Dravet syndrome.

ADDRESSING UNMET NEED: 
  • Treatment option for rare genetic condition leading ot seizures from first year of life

MECHANISM OF ACTION: Possible mechanisms include direct effects mediated through gamma-aminobutyric acid (GABA)A receptor and indirect effects involving inhibition of cytochrome P450 activity with resulting increase in blood levels of clobazam and active metabolite.

EFFICACY:
  • 2 multicenter placebo-controlled double-blind randomized studies, patients with Dravet syndrome inadequately controlled on clobazam and valproate, DIACOMIT vs placebo, n=64
  • Primary efficacy endpoint: Responder rate- patient who experienced > 50% decrease in frequency (per 30 days) of generalized clonic or tonic-clonic seizures
  • In both studies, responder rate significantly greater for DIACOMIT vs. placebo
SAFETY: 
  • Most common side effects: Somnolence, decreased appetite, agitation, ataxia, weight loss, hypotonia, nausea, tremor, dysarthria, insomnia
  • Must be dispensed with Medication Guide

LABEL


Image credits: Brainsway, Abbott, Dyax, Dompé, Biocodex 

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