Pelacarsen APOCIII LPA siRNA cardiovascular prevention

Indicated For

Elevated lipoprotein(a), atherosclerotic cardiovascular disease

About This Medication

# Pelacarsen Patient Guide: How to Get This Medication at Low or No Cost ## About Pelacarsen Pelacarsen (also known as TQJ230) is an investigational medication designed to lower lipoprotein(a) [Lp(a)], a cardiovascular risk factor. The drug works as an "antisense" medication that disrupts the production of apolipoprotein(a) in the liver, preventing your body from making new Lp(a). Early clinical studies show that pelacarsen can reduce Lp(a) levels by 67-80% compared to placebo. It is administered as a once-monthly injection under the skin that you can give yourself at home once approved. ## Current Status and Availability Pelacarsen is currently in Phase 3 clinical trials—the final stage of testing before the FDA considers it for approval. The drug is not yet available by prescription. However, Novartis Pharmaceuticals, the manufacturer, offers two pathways for patients to access pelacarsen outside of clinical trials: 1. **Managed Access Programs**: For patients with serious or life-threatening conditions who cannot participate in a clinical trial 2. **Rollover Extension Programs**: For patients who have completed certain clinical trials ## Who May Qualify for Managed Access To be considered for pelacarsen through Novartis's Managed Access Program, you must meet ALL of the following criteria: - Have a serious or life-threatening disease or condition (such as elevated Lp(a) with established cardiovascular disease) - Have no comparable or satisfactory alternative therapy available - Be unable to enroll in or continue participation in a clinical trial - Have a potential benefit from treatment that justifies the potential risks - Meet medical criteria established by Novartis medical experts - Live in a country where Managed Access provision is legally permitted ## Income Eligibility and Insurance Requirements Based on available information, Novartis has not publicly disclosed specific income thresholds or insurance requirements for the pelacarsen Managed Access Program. However, patient assistance programs typically consider: - Household income relative to federal poverty guidelines - Insurance status (uninsured, underinsured, or insured) - Out-of-pocket medication costs You should contact Novartis directly to discuss your specific financial situation and insurance coverage. ## How to Apply ### Step 1: Consult Your Physician Your licensed physician must submit an independent request on your behalf to Novartis. This is a critical first step—you cannot apply directly as a patient. ### Step 2: Gather Required Documentation While specific documentation requirements have not been publicly detailed, you should prepare: - Medical records documenting your Lp(a) levels and cardiovascular disease status - Proof of previous treatment attempts or reasons why clinical trial participation is not possible - Insurance information (if applicable) - Financial documentation if applying for financial assistance - Identification and contact information ### Step 3: Physician Submission Your physician will submit the Managed Access request to Novartis Pharmaceuticals. Contact information for inquiries: - **Manufacturer**: Novartis Pharmaceuticals - For specific contact details and submission procedures, visit Novartis's clinical trials website or ask your healthcare provider ### Step 4: Await Review Novartis will review your request based on the eligibility criteria outlined above. ## Timeline and Delivery Processing timelines for Managed Access requests have not been publicly specified. Typical review periods for compassionate use programs range from 2-4 weeks, but this may vary based on case complexity and regulatory requirements in your country. Once approved, pelacarsen will be delivered according to Novartis's standard procedures, which typically involve shipment to your home or healthcare provider. ## What If Your Application Is Denied If your Managed Access request is denied, you have several options: 1. **Ask for reconsideration**: Request that Novartis explain the denial and whether additional information could support reconsideration 2. **Explore clinical trials**: Ask your physician about enrollment in ongoing Phase 3 trials (such as the HORIZON trial or studies with background inclisiran treatment) 3. **Investigate alternative treatments**: Discuss with your cardiologist other Lp(a)-lowering options, such as lipoprotein apheresis (LA), which is currently the only FDA-approved treatment for elevated Lp(a) 4. **Contact patient advocacy organizations**: Organizations focused on cardiovascular disease may provide additional resources or guidance ## Reauthorization and Ongoing Access Information about reauthorization requirements for continued access to pelacarsen through Managed Access programs has not been publicly disclosed. You should clarify with Novartis whether your access requires periodic reapproval or if it continues as long as you remain medically eligible. ## Important Disclaimer This guide is for informational purposes only and does not constitute medical advice. Pelacarsen is an investigational drug not yet approved by the FDA. Access through Managed Access programs is limited and based on individual medical circumstances. Always consult with your healthcare provider about whether pelacarsen may be appropriate for you and to discuss the risks and benefits of any treatment. Program details, eligibility criteria, and availability may change. Contact Novartis Pharmaceuticals directly for the most current and accurate information about their patient assistance programs.

Program information last verified: March 30, 2026