Evicenter
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Aqneursa (levacetylleucine)

Ataxia-telangiectasia

Also known as IB1001
Manufacturer
IntraBio
Last updated
206 sources

Product information and disease descriptionProduct descriptionProduct information

Access and distribution

17 evidence topics · 15 sources

U.S. commercial availability

“AQNEURSA is commercially available now” (opens the source at this quote in a new tab)

“To access AQNEURSA, download and complete the Start AQNEURSA Prescription Form.” (opens the source at this quote in a new tab)

Specialty pharmacy: Curant Health

“Curant Health, a rare disease specialty pharmacy that improves health outcomes for rare and complex care patients, has been chosen by IntraBio as the exclusive specialty pharmacy for AQNEURSA™ (levacetylleucine).” (opens the source at this quote in a new tab)

“I may reply STOP to opt out of text messaging or by contacting Curant Health at 866-200-0419.” (opens the source at this quote in a new tab)

Home delivery and retail pharmacy availability

“The pharmacist will call prior to shipping the medicine to ensure that all mailing information is correct and that someone will be home to accept the delivery the next day.” (opens the source at this quote in a new tab)

“You will not be able to get or pick up AQNEURSA at a retail or neighborhood pharmacy.” (opens the source at this quote in a new tab)

“Levacetylleucine is available through the manufacturer.” (opens the source at this quote in a new tab)

Patient support program: AQNEURSA Cares

“IntraBio offers support programs to eligible patients through their Patient Support Service, AQNEURSA Cares®.” (opens the source at this quote in a new tab)

“This program includes financial support to reduce or eliminate out-of-pocket costs for qualifying patients and also connects patients with third-party resources.” (opens the source at this quote in a new tab)

“The team is available to help with individuals’ unique challenges including starting treatment, questions about taking the medication, and navigating insurance coverage.” (opens the source at this quote in a new tab)

“AQNEURSA Cares is the single point of contact for access to AQNEURSA and will work with you and your doctor to get you started on treatment.” (opens the source at this quote in a new tab)

AQNEURSA Cares services initiated by the enrollment packet
ServiceWebsite text
Benefits verification“Verification of insurance coverage for AQNEURSA”
Copay eligibility“Determination of eligibility for co-pay assistance”
Prior authorization“Support during the prior authorization process”
Shipment“Patient outreach and scheduling of medication shipments directly to the patient/caregiver”

“The complete enrollment packet consists of the Start AQNEURSA Prescription Form and the clinical information listed in the Prior Authorization Checklist.” (opens the source at this quote in a new tab)

“Fax the completed and signed Start AQNEURSA Prescription Form, along with the clinical information, to AQNEURSA Cares at 470-751-8639.” (opens the source at this quote in a new tab)

Enrollment form: prescription fields and diagnosis codes
Form itemForm text
Prescription“AQNEURSA Prescription (NDC: 83853-101-01)”
Supply“Dose & Directions (28-Day Supply)”
Diagnosis code for A-T“G11.3 Ataxia-telangiectasia”
Diagnosis code for NPC“E75.242 Niemann-Pick Disease Type C”
Body weight“Patient weight”
Eligible prescribers and patients“FOR U.S. PATIENTS AND U.S.-LICENSED PRESCRIBERS ONLY”

“Clinical documentation for Prior Authorization (see Prior Authorization Checklist)” (opens the source at this quote in a new tab)

“Copy of the front and back side of patient’s insurance card” (opens the source at this quote in a new tab)

Benefits verification and prior authorization support

“AQNEURSA Cares will contact the insurance plan to determine the patient’s out-of-pocket costs and any prior authorization requirements” (opens the source at this quote in a new tab)

“The AQNEURSA Cares team can submit a PA request if allowed by the insurance plan” (opens the source at this quote in a new tab)

“Electronic prescriptions may be submitted for refills provided by AQNEURSA Cares” (opens the source at this quote in a new tab)

Copay assistance and financial assistance

“Co-pay assistance: Patients with commercial insurance may be eligible for assistance with their co-pay from IntraBio. Restrictions apply.” (opens the source at this quote in a new tab)

“Patients who participate in Medicaid, Medicare, or any other federal healthcare program are not eligible to receive co-pay assistance.” (opens the source at this quote in a new tab)

“Patients who are unable to afford their cost-share for AQNEURSA may be eligible for financial assistance” (opens the source at this quote in a new tab)

“Co-pay assistance may lower the amount you pay for your AQNEURSA prescription to as little as $0 a month, depending on eligibility restrictions and maximum benefits.” (opens the source at this quote in a new tab)

Prior authorization checklist: diagnosis code and diagnostic information for A-T
Checklist itemChecklist text
ICD-10-CM diagnosis code“G11.3 Ataxia-Telangiectasia (A-T)”
Official descriptor of the code“Cerebellar ataxia with defective DNA repair”
Genetic confirmation“Confirmation of A-T diagnosis by genetic testing identifying biallelic pathogenic variants in the ATM gene”
Biomarker“Elevated serum alpha-fetoprotein (AFP)”
Clinical features“Observation of the clinical features of A-T”
Systemic features“oculocutaneous telangiectasia, immunodeficiency, pulmonary disease, endocrine abnormalities”
Prior authorization checklist: patient condition and medical history
Checklist itemChecklist text
Growth“Patient weight, growth, and development”
Testing“Neurological and/or psychological testing results”
Symptoms“Symptoms and quality of life (physician observation)”
Records“Chart notes”
Laboratory“Laboratory test results”
Pregnancy“Verification that the patient is not pregnant”
Hospitalization“Details on hospital admissions and discharges”
Prior authorization checklist: supporting materials and coverage disclaimer

“The following materials may help payors learn more about AQNEURSA and can be submitted with a PA or formulary exception request:” (opens the source at this quote in a new tab)

“IntraBio makes no representation or guarantee concerning coverage or reimbursement.” (opens the source at this quote in a new tab)

“Please check individual payor policies for plan-specific coverage information and requirements.” (opens the source at this quote in a new tab)

Sample letters of medical necessity and appeal for A-T

“Resources to help access an AQNEURSA prescription:” (opens the source at this quote in a new tab)

“A-T Sample Letter of Medical Necessity” (opens the source at this quote in a new tab)

“A-T Sample Letter of Appeal” (opens the source at this quote in a new tab)

Expanded access for A-T: manufacturer policy

“IntraBio currently operates an Expanded Access Program in the United States for levacetylleucine for people living with Ataxia-Telangiectasia who meet program eligibility criteria.” (opens the source at this quote in a new tab)

“Participation in the Expanded Access Program is subject to physician request, medical review, and applicable regulatory requirements and is evaluated on a case-by-case basis, as required by applicable law.” (opens the source at this quote in a new tab)

Expanded access for A-T: registry record

“This expanded access program provides investigational levacetylleucine to patients with Ataxia-Telangiectasia who are not eligible for clinical trials and have no satisfactory alternative therapies.” (opens the source at this quote in a new tab)

“Patient has a genetically confirmed diagnosis of Ataxia-Telangiectasia, a rare and serious disease with no alternative approved treatments.” (opens the source at this quote in a new tab)

“Patient weighs ≥15 kg.” (opens the source at this quote in a new tab)

“Patient resides in the United States at time of enrollment and throughout the duration of their participation in the EAP.” (opens the source at this quote in a new tab)

Published payer policies that predate the A-T approval: Cigna Healthcare

“Prior Authorization is required for benefit coverage of Aqneursa.” (opens the source at this quote in a new tab)

“Any other use is considered experimental, investigational, or unproven, including the following (this list may not be all inclusive; criteria will be updated as newly published data are available):” (opens the source at this quote in a new tab)

“A multinational, multicenter, open-label, rater-blinded prospective Phase II study is underway to assess the safety and efficacy of levacetylleucine for the treatment of Ataxia-Telangiectasia.” (opens the source at this quote in a new tab)

Policy itemPolicy text
Coverage policy number“IP0715”
Section heading“Conditions Not Covered”
Listed condition“Ataxia-Telangiectasia.”
Latest revision“Annual Revision”
Summary of changes“No criteria changes.”
Revision date“12/15/2025”
Published payer policies that predate the A-T approval: Medical Mutual
Policy itemPolicy text
Last revised date“08/21/2025”
Section heading“CONDITIONS NOT RECOMMENDED FOR APPROVAL”
Listed condition“Ataxia-Telangiectasia.”
Stated basis“Results are not yet available.”
Published payer policies that predate the A-T approval: UnitedHealthcare
Policy itemPolicy text
Program number“2026 P 2355-4”
Effective date“4/1/2026”
Diagnosis criterion“Diagnosis of Niemann-Pick disease type C (NPC)”
Authorization duration“Authorization will be issued for 12 months.”
Payer coverage policies with criteria for the A-T indication

No evidence found.