XROMI^® copay assistance program

How the copay assistance program works

1. Eligibility check. When a pharmacy processes a prescription, eligibility is automatically verified to confirm the patient is commercially insured and meets program criteria. Individuals with insurance provided by Medicaid, Medicare and other government programs are excluded.
2. eVoucher is applied. If eligible, an eVoucher is automatically generated by CoverMyMeds^®. The pharmacy applies the eVoucher at checkout to lower the patient copay to as little as $25.00.
3. Hassle-free pharmacy experience. The patient pays the reduced copay at the pharmacy; no hassle, no physical or electronic coupons to turn in.

Terms & conditions

Summary

• Eligibility: The XROMI^® eVoucher Copay Program (the “Program”)​ is available to patients with commercial insurance who meet the program’s requirements. This program is not valid for patients whose prescriptions are paid in whole or in part by Medicare, Medicaid, VA, TRICARE, or other federal or state-funded programs or where prohibited by law.
• Amount & limits: Program reduces the patient’s out-of-pocket cost to as little as $25 per prescription filled but may vary based on individual’s commercial insurance plan. Rare Disease Therapeutics, Inc. (“RDT”) reserves the right to modify the program as needed.
• Verification: Enrollment and benefit delivery are subject to verification of insurance and eligibility, clinical indication, and other program.

Full program details

1. Program Overview
The Program is a manufacturer‑sponsored patient support program offered by RDT to help eligible patients reduce their out‑of‑pocket costs for XROMI^® (hydroxyurea) oral solution when prescribed for an FDA‑approved indication.

The Program is not insurance, does not replace insurance coverage, and does not provide reimbursement directly to patients. The benefit is delivered through an electronic voucher (“eVoucher”) applied at the pharmacy at the time a qualifying prescription is processed.

2. Eligibility
A patient is eligible to participate in the Program if all of the following conditions are met:

• The patient has a valid prescription for XROMI^® written by a licensed healthcare provider in the United States for an FDA‑approved indication.
• The patient is a resident of the United States.
• The patient has commercial prescription drug insurance (non-governmental) that provides coverage for XROMI^®.

Patients whose prescriptions are paid for, in whole or in part, by any federal or state healthcare program—including but not limited to Medicare (including Part D), Medicaid, TRICARE, the Department of Veterans Affairs, or similar programs—are not eligible to participate.

The Program is subject to state-specific restrictions.

The Program is void where prohibited by law. RDT may disable benefits in jurisdictions where prohibited or limited.

3. Program Benefit
For eligible prescriptions, the Program is intended to reduce the patient’s out‑of‑pocket cost for XROMI^® to as little as $25 per prescription fill.

The actual benefit applied may vary depending on the patient’s commercial insurance plan design, including deductible status, coinsurance, and pharmacy reimbursement structure. The patient remains responsible for any applicable costs not covered by insurance or the Program.

4. Program Administration
Benefits are available only at participating pharmacies enabled for eVoucher processing. RDT is not responsible for system outages, network limitations, or non-participating pharmacies.

The eVoucher is applied electronically at participating pharmacies that are enabled to process eVouchers. No physical or digital coupon is required from the patient.

Eligibility and benefit application are subject to real‑time verification at the point of sale. RDT does not guarantee that the Program benefit will be available for every eligible prescription, including in circumstances where technical, administrative, or pharmacy system limitations prevent application.

5. Use Restrictions

• The Program is available only to individual patients and may not be transferred, sold, purchased, traded, or distributed.
• The Program may not be combined with any other manufacturer‑sponsored or third party coupon, discount, rebate, or similar offer for XROMI^®.
• The Program may not be used in a manner that causes a claim to be submitted to any federal or state healthcare program for the value of the Program benefit.
• Any prior authorization, step therapy, quantity limits, or other utilization management requirements imposed by the patient’s insurance plan remain in effect.

6. Patient Responsibilities
By participating in the Program, the patient (or the patient’s parent or legal guardian) confirms that:

• The information provided in connection with the prescription and insurance coverage is accurate and complete.
• The patient will notify the pharmacy of any changes in insurance coverage.
• The patient will comply with all applicable laws, regulations, and insurance plan requirements related to use of manufacturer assistance programs.
• Providing false or incomplete information may result in denial, termination, and recovery of benefits. Patients must notify the pharmacy and RDT of any changes in insurance coverage prior to the next fill

7. Data Use and Privacy
Information related to the patient’s prescription, insurance status, and use of the Program may be collected and used by RDT and its service providers solely for purposes of administering the Program, verifying eligibility, and meeting legal and regulatory obligations.

All information will be handled in accordance with applicable privacy laws (such as HIPAA) and RDT’s privacy practices.

8. Modification or Termination
RDT reserves the right to modify, suspend, or terminate the Program, in whole or in part, at any time and for any reason, without notice. Changes may include eligibility criteria, benefit structure, or Program duration. Continued participation after any modification constitutes acceptance of the updated terms.

9. No Guarantee of Coverage
Participation in the Program does not guarantee insurance coverage or reimbursement for XROMI^®. Coverage determinations are made solely by the patient’s insurance plan.

10. Governing Law
These Terms & Conditions are governed by the laws of the United States and the state in which the patient resides, to the extent not preempted by federal law.

By using the XROMI^® eVoucher Copay Program, the patient acknowledges that they have read, understand, and agree to these Terms & Conditions.

² Actual amounts of copay reimbursement may vary.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS
XROMI is contraindicated in patients who have demonstrated a previous hypersensitivity to hydroxyurea or any other component of its formulation.

WARNINGS AND PRECAUTIONS
Myelosuppression
Hydroxyurea causes severe myelosuppression. Do not initiate treatment with hydroxyurea in patients if bone marrow function is markedly depressed. Bone marrow suppression may occur, and leukopenia is generally its first and most common manifestation. Thrombocytopenia and anemia occur less often and are seldom seen without a preceding leukopenia.

Some patients, treated at the recommended initial dose of 15 mg/kg/day, have experienced severe or life-threatening myelosuppression.

Evaluate hematologic status prior to and during treatment with XROMI. Provide supportive care and modify dose or discontinue XROMI as needed. Recovery from myelosuppression is usually rapid when therapy is interrupted.

Hemolytic Anemia
Cases of hemolytic anemia in patients treated with hydroxyurea for myeloproliferative diseases have been reported. In the setting of confirmed diagnosis of hemolytic anemia and in the absence of other causes, discontinue XROMI.

Malignancies
Hydroxyurea is a human carcinogen. In patients receiving long-term hydroxyurea for myeloproliferative disorders (a condition for which XROMI is not approved), secondary leukemia has been reported.

Secondary leukemia has also been reported in patients treated with long-term hydroxyurea for sickle cell anemia. Leukemia has also been reported in patients with sickle cell anemia and no prior history of treatment with hydroxyurea.

All patients using XROMI should be followed up on a long-term basis with regular blood counts to detect development of leukemia.

Skin cancer has also been reported in patients receiving long-term hydroxyurea. Advise protection from sun exposure and monitor for the development of secondary malignancies.

Embryo-Fetal Toxicity
Based on the mechanism of action and findings in animals, XROMI can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus and to avoid becoming pregnant while being treated with XROMI.

Advise females of reproductive potential to use effective contraception during and after treatment with XROMI for at least 6 months after therapy. Advise males of reproductive potential to use effective contraception during and after treatment with XROMI for at least 1 year after therapy.

Vasculitic Toxicities
Cutaneous vasculitic toxicities, including vasculitic ulcerations and gangrene, have occurred in patients with myeloproliferative disorders during therapy with hydroxyurea. If cutaneous vasculitic ulcers occur, institute treatment and discontinue XROMI.

Live Vaccines
Avoid use of live vaccines in patients taking XROMI as it may potentiate the replication of the virus and/or may increase the adverse reaction of the vaccine because normal defense mechanisms may be suppressed by XROMI. Vaccination with live vaccines in a patient receiving XROMI may result in severe infection. Patient’s antibody response to vaccines may be decreased. Consider consultation with a specialist.

Risks with Concomitant Use of Antiretroviral Drugs
Pancreatitis, hepatotoxicity, and peripheral neuropathy have occurred when hydroxyurea was administered concomitantly with antiretroviral drugs, including didanosine and stavudine.

Macrocytosis
XROMI may cause macrocytosis, which is self-limiting, and is often seen early in the course of treatment. The morphologic change resembles pernicious anemia, but is not related to vitamin B₁₂ or folic acid deficiency. This may mask the diagnosis of pernicious anemia. Prophylactic administration of folic acid is recommended.

Laboratory Test Interference
Interference with uric acid, urea, or lactic acid assays is possible, rendering falsely elevated results of these in patients treated with hydroxyurea.

Hydroxyurea may falsely elevate sensor glucose results from certain continuous glucose monitoring (CGM) systems and may lead to hypoglycemia if sensor glucose results are relied upon to dose insulin.

ADVERSE REACTIONS
Most common adverse reactions (incidence >5%) are neutropenia, thrombocytopenia, and papular rash.

USE IN SPECIFIC POPULATIONS

• Females and Males of Reproductive Potential: XROMI can cause fetal harm when administered to a pregnant woman. Verify the pregnancy status of females of reproductive potential prior to initiating XROMI therapy. Advise females to immediately report pregnancy. XROMI may damage spermatozoa and testicular tissue, resulting in possible genetic abnormalities. Based on findings in animals and humans, male fertility may be compromised by treatment with XROMI.
• Pediatric Use: Continuous follow-up of the growth of treated children is recommended.
• Renal Impairment: Reduce dosage and closely monitor the hematologic parameters when XROMI is administered to patients with renal impairment who have a creatinine clearance of less than 60 mL/min.
• Hepatic Impairment: Close monitoring of hematologic parameters is advised in these patients receiving XROMI.

OVERDOSAGE
Acute mucocutaneous toxicity has been reported in patients receiving hydroxyurea at doses several times above the therapeutic dose. Soreness, violet erythema, oedema on palms and soles followed by scaling of hand and feet, severe generalized hyperpigmentation of the skin, and stomatitis have been observed.

Please see complete Prescribing Information including BOXED WARNING and Patient Information or at www.xromi.com/pi.

To report SUSPECTED ADVERSE REACTIONS, contact Rare Disease Therapeutics, Inc., at 1-844-472-7389 or by email at safety@raretx.com or contact FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

XRO-ISIF-005