Marketing Authorizations: EndeavorRX digital therapy for ADHD, FINTEPLA for Dravet Syndrome, GALLANT defibrillator systems
EndeavorRx digital therapy
INDICATION FOR USE: Digital therapeutic indicated to improve attention function as measured by computerbased testing in children ages 8-12 years old with primarily inattentive or combined-type ADHD, who have a demonstrated attention issue. Patients who engage with EndeavorRx demonstrate improvements in a digitally assessed measure Tests of Variables of Attention (TOVA) of sustained and selective attention and may not display benefits in typical behavioral symptoms, such as hyperactivity. EndeavorRx should be considered for use as part of a therapeutic program that may include: clinician-directed therapy, medication, and/or educational programs, which further address symptoms of the disorder.
DEVICE TYPE: Digital therapy device for Attention Deficit Hyperactivity Disorder. Software intended to provide therapy for ADHD or any of its individual symptoms as an adjunct to clinician supervised treatment.
ADDRESSING UNMET NEED: First digital therapeutic intended to improve symptoms associated with ADHD. First game-based therapeutic granted marketing authorization by the FDA for any type of condition.
PERFORMANCE & SAFETY:
- Multiple studies in >600 children
- Evaluated demonstrated improvements in attention function, as measured by TOVA, academic performance measures, and other assessment tools
- No serious adverse events reported
- Most common adverse events: Frustration, headache, dizziness, emotional reaction, and aggression
REGULATORY PATHWAY: De Novo classification
FINTEPLA (fenfluramine) oral solution
INDICATION: Treatment of seizures associated with Dravet syndrome in patients 2 years of age and older
MECHANISM OF ACTION: Exact mechanism for treatment of seizures unknown;
fenfluramine and metabolite, norfenfluramine, increase extracellular levels of serotonin and exhibit agonist activity at serotonin 5HT-2 receptors
ADDRESSING UNMET NEED: New therapy for Dravet syndrome- a life-threatening, rare and chronic form of epilepsy that is characterized by severe and unrelenting seizures despite medical treatment
EFFICACY & SAFETY:
- Two clinical studies, n=202 subjects 2-18 years old; Fintepla vs placebo
- Primary endpoint: Change from baseline in the frequency of convulsive seizures
- Significantly greater reductions in convulsive seizures frequency with Fintepla within 3-4 weeks, remained consistent over the 14- to 15-week treatment periods
- Boxed warning: Valvular heart disease (VHD) and pulmonary arterial hypertension (PAH)
- Most common adverse reactions: Decreased appetite; drowsiness, sedation and lethargy; diarrhea; constipation; abnormal echocardiogram; fatigue or lack of energy; ataxia, balance disorder, gait disturbance, blood pressure; drooling, salivary hypersecretion, pyrexia; upper respiratory tract infection; vomiting; decreased weight; risk of falls; and status epilepticus.
REGULATORY PATHWAY: NDA
- Priority Review, Orphan Drug Designation
- Schedule IV controlled substance
- Restricted drug distribution program, REMS)
- Special certification to dispense, cardiac monitoring with echocardiograms
GALLANT implantable cardioverter defibrillator (ICD) and cardiac resynchronization therapy defibrillator (CRT-D) devices
INDICATION FOR USE: For automated treatment of life-threatening ventricular arrhythmias; also indicated to treat symptoms in patients who have congestive heart failure with ventricular dyssynchrony. In addition, dual chamber ICD and CRT-D devices with the AT/AF detection algorithm are indicated in patients with atrial tachyarrhythmias or those patients who are at significant risk of developing atrial tachyarrhythmias.
MR Conditional ICDs and CRT-Ds are conditionally safe for use in the MRI environment when used in a complete MR Conditional system and according to instructions in the MRI-Ready Systems manual. Scanning under different conditions may result in severe patient injury, death or device malfunction.
The myMerlinPulse™ mobile application is indicated for use by patients with supported Abbott Medical implanted heart devices.
- Implantable Cardioverter Defibrillator (ICD) and Cardiac Resynchronization Therapy Defibrillator (CRT-D) device intended to provide ventricular antitachycardia pacing and ventricular cardioversion/defibrillation; also intended to resynchronize right and left ventricles
- Pairs with Abbott’s secure myMerlinPulse™, an iOS- and Android™- compatible mobile smartphone app that helps streamline communication between doctors and their patients
- App provides people with access to data, device performance, and transmission history, which helps them take an active role in their healthcare
- Through the app, physicians can continuously monitor their patients remotely allowing for identification of asymptomatic episodes as well as patient-triggered transmissions, which can lead to earlier intervention and reduce clinical burden
ADDRESSING UNMET NEED:
- 6.1 million people in US with cardiac arrhythmias, or abnormal heart rhythms
- ICDs are used to help reduce the risks of life-threatening arrhythmias.
- CRT-Ds can be used to restore the heart’s natural pattern of beating for patients with heart failure or in situations when the heart’s chambers beat out of sync
REGULATORY PATHWAY: PMA
Image credits: Akili, Zogenix, Abbott