DUVYZAT resources for you and your practice

DUVYZAT was approved by the US Food and Drug Administration on March 21, 2024.

DUVYZAT is indicated for all patients with Duchenne muscular dystrophy 6 years of age and older, regardless of genetic mutation or ambulatory status.^1,* *The EPIDYS clinical trial did not include nonambulatory patients.

DUVYZAT is a histone deacetylase inhibitor indicated for the treatment of Duchenne muscular dystrophy in patients 6 years of age and older in the US.¹ DUVYZAT is not approved for patients with Becker muscular dystrophy (BMD). We recognize the significant unmet medical needs of people living with BMD and their families. Our program for BMD is in the early stages of clinical development. It is important to note that this question pertains to an unapproved or uncleared use of DUVYZAT. For further details, please contact Medical Information at medinfo@itftherapeutics.com.

ITF Therapeutics is the US-based rare disease division of Italfarmaco S.p.A. Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development of many innovative therapeutic products approved for use by patients around the world. The company operates in more than 90 countries on 5 continents and continues to advance promising research to address unmet medical needs in a wide range of therapeutic areas. In January 2024, Italfarmaco launched ITF Therapeutics as a new division in the United States with a focus on the development and commercialization of products to treat rare diseases, including Duchenne muscular dystrophy. Building on a legacy grounded in collaboration and innovation, ITF Therapeutics strives to partner with leaders from the US patient advocacy and treatment communities to ensure that our programs reflect and support their unique needs and goals. The establishment of ITF Therapeutics also reflects Italfarmaco's goal to build a world-class team of experts who share a passion to make a positive impact for rare disease communities. For more information about ITF Therapeutics, please visit itftherapeutics.com.

We are committed to making treatment with DUVYZAT available to as many appropriate patients in the US as possible by working with payers to enable access. We also offer a variety of programs to help patients access treatment while insurers complete their assessments. These include:

• A copay assistance program for commercially insured patients, where patients may pay as little as $0
• A temporary supply program that provides a limited supply of DUVYZAT to help ensure a timely start for appropriate patients facing insurance-related delays
• A temporary supply program for patients currently on therapy who may be changing insurance providers and might otherwise experience a gap in treatment
• A patient assistance program providing DUVYZAT at no charge for eligible patients, based on established criteria

We understand that patients may encounter out-of-pocket costs in situations where insurance coverage is insufficient. As a reflection of our commitment to making treatment with DUVYZAT available to as many appropriate patients in the US as possible, we have created a suite of programs to help address potential financial concerns, including:

• A copay assistance program for commercially insured patients, where patients may pay as little as $0
• A temporary supply program that provides a limited supply of DUVYZAT to help ensure a timely start for appropriate patients facing insurance-related delays
• A temporary supply program for patients currently on therapy who may be changing insurance providers and might otherwise experience a gap in treatment
• A patient assistance program providing DUVYZAT at no charge for eligible patients, based on established criteria

For patients currently enrolled in ITF ARC: An additional In-Home Lab Monitoring Program Form is required to initiate in-home laboratory monitoring. For new patients starting treatment with DUVYZAT: Please complete the ITF ARC Patient Start Form and In-Home Lab Monitoring Program Form. Both forms can be faxed or mailed to PANTHERx. *The DUVYZAT In-Home Lab Monitoring Program is only available in each US state and the District of Columbia. Program rules apply.

Patients should fast prior to testing if triglycerides are included in the laboratory panel.

Naven Health will fax the results to your office within 24 hours of the lab’s reporting.

If a patient misses a scheduled lab draw, and Naven Health isn't able to reschedule their appointment within 3 calendar days, PANTHERx will notify you. Together, you'll determine whether the visit should be rescheduled within the appropriate time frame or skipped in favor of the next scheduled visit. Naven Health will make every effort to reschedule the appointment with the patient or caregiver, when applicable.

Gastrointestinal disturbances, including diarrhea, nausea/vomiting, and abdominal pain, were common adverse reactions in DUVYZAT clinical trials in Duchenne muscular dystrophy. Antiemetics or antidiarrheal medications may be considered during treatment with DUVYZAT. Fluid and electrolytes should be replaced as needed to prevent dehydration.

Please notify PANTHERx of any dose modifications so the patient’s treatment record can be updated accordingly. The laboratory monitoring schedule should then be adjusted based on the clinical rationale for the dose change.

Once a completed referral, including patient monitoring consent, has been received from PANTHERx, Naven Health will contact the patient or caregiver within 24 hours for an introductory call. They will confirm the patient's start date of the lab draw schedule. Naven Health will schedule subsequent appointments directly with the patient or caregiver.

If a critical lab value (CLV) occurs, PANTHERx will inform you within 24 hours. If a critically low value is reported for a patient due for refill, PANTHERx will hold the DUVYZAT shipment until they receive further instruction from you.

Lab monitoring should follow the schedule outlined in the Prescribing Information. Limited adjustments to the monitoring protocol may be possible in select situations. Please indicate any requested modifications on the monitoring enrollment form and/or call PANTHERx directly to discuss.

Lab samples dropped off at Labcorp on the same day as sample collection may be ready as soon as the next day. For lab samples that require shipping via FedEx, results turnaround time is 2 to 4 business days.