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ph-1122

Wakix (pitolisant) Prior Authorization with Quantity Limit Program Summary

Policy Number: PH-1122

This program applies to Blue Partner, Commercial, GenPlus, NetResults A series, SourceRx and Health Insurance Marketplace formularies.

POLICY REVIEW CYCLE

Effective Date	Date of Origin
04-01-2025	04-01-2020

FDA LABELED INDICATIONS AND DOSAGE

Agent(s)	FDA Indication(s)	Notes	Ref#
Wakix® (pitolisant) Tablet	Treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy Treatment of excessive daytime sleepiness in pediatric patients 6 years of age and older with narcolepsy		1

Agent(s)

FDA Indication(s)

Notes

Ref#

Wakix®

(pitolisant)

Tablet

Treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy

Treatment of excessive daytime sleepiness in pediatric patients 6 years of age and older with narcolepsy

See package insert for FDA prescribing information: https://dailymed.nlm.nih.gov/dailymed/index.cfm

CLINICAL RATIONALE

Narcolepsy

Narcolepsy is a chronic neurological disorder that affects the brain's ability to regulate sleep-wake cycles. At various times throughout the day, patients with narcolepsy experience irresistible bouts of sleep and could fall asleep. If left undiagnosed or untreated, narcolepsy can interfere with psychological, social, and cognitive function and development and can inhibit academic, work, and social activities.(2) Symptoms may include excessive daytime sleepiness (EDS), cataplexy, sleep paralysis, and hallucinations. All patients diagnosed with narcolepsy will have excessive daytime sleepiness. However, sleepiness in narcolepsy is more like a “sleep attack”, where an overwhelming sense of sleepiness comes on quickly.(2) The American Family Physician recommends referral to a sleep clinic if narcolepsy is suspected.(3) The American Academy of Sleep Medicine (AASM) indicates treatment goals should be to alleviate daytime sleepiness and produce the fullest possible return of normal function for patients at work, school, home, and socially.(4)

Excessive daytime sleepiness (EDS) is characterized by persistent sleepiness regardless of how much sleep an individual gets at night. In between sleep attacks, individuals have normal levels of alertness, particularly if doing activities that keep their attention. The most common causes of EDS include narcolepsy, obstructive sleep apnea, shift work disorder, sleep deprivation, medication effects, and other medical and psychiatric conditions.(5)

Narcolepsy has two types, narcolepsy with cataplexy and without cataplexy. Narcolepsy with cataplexy involves the sudden loss of voluntary muscle tone while awake. It is often triggered by sudden, strong emotions such as laughter, fear, anger, stress, or excitement. The symptoms of cataplexy may appear weeks or even years after the onset of EDS.(2)

AASM 2021 guidelines combined the recommendations for narcolepsy with cataplexy and EDS associated with narcolepsy. The AASM recommend the following for the pharmacologic treatment of adults with narcolepsy:(6)

Strong treatment recommendations:
- Modafinil
- Pitolisant
- Sodium oxybate
- Solriamfetol
Conditional treatment recommendations:
- Armodafinil
- Dextroamphetamine
- Methylphenidate
There was insufficient evidence to make recommendations for SSRI and SNRIs for the treatment of narcolepsy.(6)

AASM 2021 guidelines combined the recommendations for EDS associated with narcolepsy. The AASM recommend the following for the pharmacologic treatment of pediatric patients with narcolepsy:(6)

Conditional treatment recommendations:
- Modafinil
- Sodium Oxybate (6)

Efficacy

Excessive Daytime Sleepiness (EDS) in Patients with Narcolepsy

The efficacy of Wakix for the treatment of excessive daytime sleepiness in adult patients with narcolepsy was evaluated in two multicenter, randomized, double-blind, placebo-controlled studies (Study 1; NCT01067222 and Study 2; NCT01638403). EDS was assessed using the ESS, an 8-item questionnaire by which patients rate their perceived likelihood of falling asleep during usual daily life activities.(1)

Wakix demonstrated statistically significantly greater improvement on the primary endpoint, the least square mean final ESS score compared to placebo.(1)

The efficacy of Wakix for the treatment of excessive daytime sleepiness in pediatric patients 6 years of age and older with narcolepsy was evaluated in one multicenter, randomized, double-blind, placebo-controlled study (Study 4; NCT02611687).(1) Pediatric patients 6 to 17 years who met the International Classification of Sleep Disorders (ICSD-3) criteria for narcolepsy and who had a Pediatric Daytime Sleepiness Scale (PDSS) score > 15 were eligible to enroll in the study. Patients were to have discontinued psychostimulants at least 14 days prior to enrollment.(7)

WAKIX demonstrated statistically significantly greater improvement on the least square mean change from baseline to the end of treatment in final (PDSS) total score compared to placebo, of -3.41 points (95% CI: -5.52, -1.31). Study 4 included global assessments, which showed positive trends supporting PDSS total score of improvement in favor of WAKIX. (1)

Cataplexy in Patients with Narcolepsy

The efficacy of Wakix for the treatment of cataplexy in adult patients with narcolepsy was evaluated in two multicenter, randomized, double-blind, placebo-controlled studies (Study 3; NCT01800045 and Study 1; NCT01067222).(1)

Wakix demonstrated statistically significantly greater improvement on the primary endpoint, the change in geometric mean number of cataplexy attacks per week from baseline to the average of the 4-week stable dosing period for Wakix compared to placebo.(1)

Safety

Wakix has the following contraindications for use:

Patients with severe hepatic impairment
Known hypersensitivity to pitolisant or any component of the formulation(1)

REFERENCES

Number	Reference
1	Wakix prescribing Information. Harmony Biosciences, LLC. June 2024.
2	National Institute of Neurological Disorders and Stroke. Narcolepsy. NIH Publication No. 17-1637.
3	Ramar, Kannan MD and Olson, Eric MD. Management of Common Sleep Disorders. Am Fam Physician. 2013 Aug 15; 88(4): 231-238.
4	Krahn, Lois MD, et al. Quality Measures for the Care of Patients with Narcolepsy. Journal of Clinical Sleep Medicine. 2015; Vol. 11(3).
5	Pagel J. Excessive daytime sleepiness. Am Fam Physician. 2009;79(5): 391-395.
6	Maski K, Trotti LM, Kotagal S, et al. Treatment of central disorders of hypersomnolence: an American Academy of Sleep Medicine clinical practice guideline. J Clin Sleep Med. 2021;17(9):1881-1893.
7	Dauvilliers Y, Lecendreux M, Lammers GJ, Franco P, et al. Safety and efficacy of pitolisant in children aged 6 years or older with narcolepsy with or without cataplexy. Lancet Neurol. 2023 Apr;22(4):303-311

POLICY AGENT SUMMARY PRIOR AUTHORIZATION

Target Brand Agent(s)	Target Generic Agent(s)	Strength	Targeted MSC	Available MSC	Final Age Limit	Preferred Status

Wakix	pitolisant hcl tab	17.8 MG ; 4.45 MG	M ; N ; O ; Y	N

POLICY AGENT SUMMARY QUANTITY LIMIT

Target Brand Agent Name(s)	Target Generic Agent Name(s)	Strength	QL Amount	Dose Form	Day Supply	Duration	Addtl QL Info	Allowed Exceptions	Targeted NDCs When Exclusions Exist

Wakix	pitolisant hcl tab	17.8 MG ; 4.45 MG	60	Tablets	30	DAYS

CLIENT SUMMARY – PRIOR AUTHORIZATION

Target Brand Agent Name(s)	Target Generic Agent Name(s)	Strength	Client Formulary
Wakix	pitolisant hcl tab	17.8 MG ; 4.45 MG	Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx

CLIENT SUMMARY – QUANTITY LIMITS

Target Brand Agent Name(s)	Target Generic Agent Name(s)	Strength	Client Formulary
Wakix	pitolisant hcl tab	17.8 MG ; 4.45 MG	Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx

PRIOR AUTHORIZATION CLINICAL CRITERIA FOR APPROVAL

Module	Clinical Criteria for Approval
	Initial Evaluation Target Agent(s) will be approved when ALL of the following are met: ONE of the following: The patient is 6 years of age or greater with a diagnosis of excessive daytime sleepiness associated with narcolepsy OR The patient is an adult with excessive daytime sleepiness or cataplexy associated with narcolepsy AND ONE of the following: The patient has tried and had an inadequate response to armodafinil OR modafinil OR The patient has an intolerance or hypersensitivity to armodafinil OR modafinil OR The patient has an FDA labeled contraindication to BOTH armodafinil AND modafinil OR The patient has been prescribed the requested non-controlled agent due to comorbid conditions OR concerns about controlled substance use AND If the patient has an FDA labeled indication, then ONE of the following: The patient’s age is within FDA labeling for the requested indication for the requested agent OR The prescriber has provided information in support of using the requested agent for the patient’s age for the requested indication AND The prescriber is a specialist in the area of the patient’s diagnosis (e.g., neurologist, psychiatrist, sleep disorder specialist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND The patient does NOT have any FDA labeled contraindications to the requested agent Length of Approval: 12 months Step therapy requirement may not apply if a prior health plan paid for the medication - documentation of a paid claim may be required. Renewal Evaluation* Target Agent(s) will be approved when ALL of the following are met: The patient has been previously approved for the requested agent through the plan’s Prior Authorization process [Note: patients not previously approved for the requested agent will require initial evaluation review] AND The patient has had clinical benefit with the requested agent AND The prescriber is a specialist in the area of the patient’s diagnosis (e.g., neurologist, psychiatrist, sleep disorder specialist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND The patient does NOT have any FDA labeled contraindications to the requested agent Length of Approval: 12 months Note: If Quantity Limit applies, please refer to Quantity Limit Criteria.

Module

Clinical Criteria for Approval

Initial Evaluation

Target Agent(s) will be approved when ALL of the following are met:

ONE of the following:
1. The patient is 6 years of age or greater with a diagnosis of excessive daytime sleepiness associated with narcolepsy OR
2. The patient is an adult with excessive daytime sleepiness or cataplexy associated with narcolepsy AND
ONE of the following:
1. The patient has tried and had an inadequate response to armodafinil OR modafinil OR
2. The patient has an intolerance or hypersensitivity to armodafinil OR modafinil OR
3. The patient has an FDA labeled contraindication to BOTH armodafinil AND modafinil OR
4. The patient has been prescribed the requested non-controlled agent due to comorbid conditions OR concerns about controlled substance use AND
If the patient has an FDA labeled indication, then ONE of the following:
1. The patient’s age is within FDA labeling for the requested indication for the requested agent OR
2. The prescriber has provided information in support of using the requested agent for the patient’s age for the requested indication AND
The prescriber is a specialist in the area of the patient’s diagnosis (e.g., neurologist, psychiatrist, sleep disorder specialist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND
The patient does NOT have any FDA labeled contraindications to the requested agent

Length of Approval: 12 months

*Step therapy requirement may not apply if a prior health plan paid for the medication - documentation of a paid claim may be required.

Renewal Evaluation

Target Agent(s) will be approved when ALL of the following are met:

The patient has been previously approved for the requested agent through the plan’s Prior Authorization process [Note: patients not previously approved for the requested agent will require initial evaluation review] AND
The patient has had clinical benefit with the requested agent AND
The prescriber is a specialist in the area of the patient’s diagnosis (e.g., neurologist, psychiatrist, sleep disorder specialist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis AND
The patient does NOT have any FDA labeled contraindications to the requested agent

Length of Approval: 12 months

Note: If Quantity Limit applies, please refer to Quantity Limit Criteria.

QUANTITY LIMIT CLINICAL CRITERIA FOR APPROVAL

Module	Clinical Criteria for Approval
QL with PA	Quantity limit for the Target Agent(s) will be approved when ONE of the following is met: The requested quantity (dose) does NOT exceed the program quantity limit OR The requested quantity (dose) exceeds the program quantity limit AND ONE of the following: BOTH of the following: The requested agent does NOT have a maximum FDA labeled dose for the requested indication AND There is support for therapy with a higher dose for the requested indication OR BOTH of the following: The requested quantity (dose) does NOT exceed the maximum FDA labeled dose for the requested indication AND There is support for why the requested quantity (dose) cannot be achieved with a lower quantity of a higher strength that does NOT exceed the program quantity limit OR BOTH of the following: The requested quantity (dose) exceeds the maximum FDA labeled dose for the requested indication AND There is support for therapy with a higher dose for the requested indication Length of Approval: up to 12 months

Module

Clinical Criteria for Approval

QL with PA

Quantity limit for the Target Agent(s) will be approved when ONE of the following is met:

The requested quantity (dose) does NOT exceed the program quantity limit OR
The requested quantity (dose) exceeds the program quantity limit AND ONE of the following:
1. BOTH of the following:
  1. The requested agent does NOT have a maximum FDA labeled dose for the requested indication AND
  2. There is support for therapy with a higher dose for the requested indication OR
2. BOTH of the following:
  1. The requested quantity (dose) does NOT exceed the maximum FDA labeled dose for the requested indication AND
  2. There is support for why the requested quantity (dose) cannot be achieved with a lower quantity of a higher strength that does NOT exceed the program quantity limit OR
3. BOTH of the following:
  1. The requested quantity (dose) exceeds the maximum FDA labeled dose for the requested indication AND
  2. There is support for therapy with a higher dose for the requested indication

Length of Approval: up to 12 months

This pharmacy policy is not an authorization, certification, explanation of benefits or a contract. Eligibility and benefits are determined on a case-by-case basis according to the terms of the member’s plan in effect as of the date services are rendered. All pharmacy policies are based on (i) information in FDA approved package inserts (and black box warning, alerts, or other information disseminated by the FDA as applicable); (ii) research of current medical and pharmacy literature; and/or (iii) review of common medical practices in the treatment and diagnosis of disease as of the date hereof. Physicians and other providers are solely responsible for all aspects of medical care and treatment, including the type, quality, and levels of care and treatment.
The purpose of Blue Cross and Blue Shield of Alabama’s pharmacy policies are to provide a guide to coverage. Pharmacy policies are not intended to dictate to physicians how to practice medicine. Physicians should exercise their medical judgment in providing the care they feel is most appropriate for their patients.
Neither this policy, nor the successful adjudication of a pharmacy claim, is guarantee of payment.