Patient Experience

Navigating AHP: Patient experiences with GIVLAARI^1,2

Acute hepatic porphyria (AHP) causes severe and debilitating attacks that can disrupt and overwhelm the lives of patients with the disease. GIVLAARI® (givosiran) may help reduce AHP attacks in adults.^3,4

Mike and Amalia are real patients diagnosed with AHP and taking GIVLAARI. Amalia is an Alnylam Patient Ambassador taking GIVLAARI. Explore their profiles and discover their unique journeys with the disease. Below, you will also find helpful resources for your patients who are considering treatment with GIVLAARI.

Mike’s Journey

"I would describe porphyria as a thief. It has taken everything from me."

Mike is a working father of two whose long battle and eventual diagnosis with acute intermittent porphyria (AIP) changed the trajectory of his life.

+SEE AMALIA'S JOURNEY

History with AHP

Pre-diagnosis

Began having mild attacks that became significantly more aggressive over time
- Attacks initially felt like heartburn and progressed into severe abdominal pain
Cholecystectomy performed
Tested for Crohn's disease and appendicitis
Misdiagnosed with prostatitis and an intestinal infection
Labeled as "drug seeking" at the pain clinic

Post-diagnosis

Diagnosed with AIP 4 years after first attack
Visited ER every 3-6 weeks due to attacks
Prescription pain medications

Amalia is an Alnylam Patient Ambassador taking GIVLAARI

Amalia's Journey

"The reason I stay on GIVLAARI is because with fewer attacks, I feel like I have more control back in my life. I’m able to make plans for the future."

Amalia had a lot of goals and ambitions as she started her freshman year of college. A year later, she was diagnosed with acute intermittent porphyria (AIP) and had to put her dream of helping others on hold to focus on her health.

Amalia is an Alnylam Patient Ambassador taking GIVLAARI

Real GIVLAARI® patient Mike's Journey with AIP

+SEE MIKE'S JOURNEY

History with AHP

Pre-diagnosis

Experienced childhood allergies to certain foods and materials
Began having mild attacks that became significantly more aggressive over time
- Attacks initially felt like nausea and progressed into severe abdominal pain
Visited emergency room several times without receiving any diagnosis
Tested for appendix and gallbladder issues
Was refused pain medication by emergency room

Post-diagnosis

Diagnosed with AIP after finding out it was genetic
Avoided managing disease until attacks became too severe
Started seeing therapist due to anxiety
Visited hospital every month for cyclical attacks

Life with AHP Today

Treatment

GIVLAARI – Once-monthly subcutaneous injection⁴

Day-to-day living with AIP

Experienced a reduction in attacks
Monitors/avoids common attack triggers (alcohol, smoking, certain medications, etc)^5,6
Has occasional fatigue and nausea during menstrual cycle

Individual results may vary.

"The reason I stay on GIVLAARI is because with fewer attacks, I feel like I have more control back in my life. I’m able to make plans for the future." –Amalia

See GIVLAARI clinical data and how it may help your patients

Resources for your patients

Alnylam is committed to providing disease and product information to patients considering or currently taking GIVLAARI. Learn how your patients can get access to educational resources.

Patient Support Through Alnylam Assist^®

Alnylam Assist^®offers support services to guide you and your patients through treatment with GIVLAARI, including:

GIVLAARI coverage, prior authorization, coding, reimbursement education, and patient-specific benefit verification
Low or no-cost programs for eligible patients,* including copay support and a Patient Assistance Program (PAP)
One-on-one support for you and your patients throughout their treatment journey from Case Managers, Field Reimbursement Directors, and Patient Education Liaisons (PELs)

*Patients must meet specified eligibility criteria to qualify for assistance. Alnylam reserves the right to make eligibility determinations and to modify or discontinue any program at any time.

Alnylam Case Managers

An Alnylam Case Manager is available to support your patient one-on-one once a Start Form has been submitted. They will connect with your patient and your practice within 2 business days to help with treatment initiation.

Support services include:

Benefit verification and a Summary of Benefits readout specific to your patient
Financial assistance eligibility assessment and enrollment^†
Support with continuity of care (through coverage changes, travel plans, etc)
Patient outreach before and after each treatment
Coordination with other Alnylam Assist® Support Team members

^†Patients must meet specific eligibility criteria to qualify for assistance. Alnylam reserves the right to make eligibility determinations and to modify or discontinue any program at any time.

Alnylam Patient Education Liaisons (PELs) icon

Alnylam PELs

PELs have backgrounds in nursing and are available to provide education to patients and their families throughout the treatment journey.

Support services include:

Providing pre- and post-treatment disease education
Answering questions about treatment
Hosting individual, family, or group meetings
Connecting patients to other resources and support

Find Out More About Alnylam Assist^®

Materials to help your patients along the way

The Patient Welcome Kit provides a number of helpful resources for your patients who are getting started on GIVLAARI:

Starting Your Journey Brochure – A guide to starting treatment with GIVLAARI

GIVLAARI Treatment Journal – For patients to keep handy as they continue treatment. It can help them keep track of important milestones and learn about ways to potentially manage side effects with their doctor

Alnylam Assist^® Brochure – Helps patients get access to GIVLAARI and describes patient support services

Notebook & Pen – For patients to track their treatment journey

Answer your patients' frequently asked questions

Your patients may have questions about treatment with GIVLAARI. Get answers to common questions that patients may ask when considering or taking GIVLAARI.

Explore Patient FAQs

Do you have more questions about GIVLAARI?

Schedule an informative discussion with a GIVLAARI Representative.

Contact a Representative

NEXT: Access & Support

References: 1. Simon A, Pompilus F, Querbes W, et al. Patient. 2018;11(5):527-537. 2. Kubisch I, Wohmann N, Wissniowski TT, et al. J Clin Med. 2024;13(22):6779. 3. Anderson KE, Bloomer JR, Bonkovsky HL, et al. Ann Intern Med. 2005;142:439-451. 4. GIVLAARI [prescribing information]. Cambridge, MA: Alnylam Pharmaceuticals, Inc. 5. Balwani M, Wang B, Anderson KE, et al; Porphyrias Consortium of the Rare Diseases Clinical Research Network. Hepatology. 2017;66:1314-1322. 6. Lin CS-Y, Lee M-J, Park SB, Kiernan MC. Clin Neurophysiol. 2011;122:2336-2344.

IMPORTANT SAFETY INFORMATION

Contraindications

GIVLAARI® (givosiran) is contraindicated in patients with known severe hypersensitivity to givosiran. Reactions have included anaphylaxis.

Anaphylactic Reaction

Anaphylaxis has occurred with GIVLAARI treatment (<1% of patients in clinical trials). Ensure that medical support is available to appropriately manage anaphylactic reactions when administering GIVLAARI. Monitor for signs and symptoms of anaphylaxis. If anaphylaxis occurs, immediately discontinue administration of GIVLAARI and institute appropriate medical treatment.

Hepatic Toxicity

Transaminase elevations (ALT) of at least 3 times the upper limit of normal (ULN) were observed in 15% of patients receiving GIVLAARI in the placebo-controlled trial. Transaminase elevations primarily occurred between 3 to 5 months following initiation of treatment.

Measure liver function tests prior to initiating treatment with GIVLAARI, repeat every month during the first 6 months of treatment, and as clinically indicated thereafter. Interrupt or discontinue treatment with GIVLAARI for severe or clinically significant transaminase elevations. In patients who have dose interruption and subsequent improvement, reduce the dose to 1.25 mg/kg once monthly. The dose may be increased to the recommended dose of 2.5 mg/kg once monthly if there is no recurrence of severe or clinically significant transaminase elevations at the 1.25 mg/kg dose.

Renal Toxicity

Increases in serum creatinine levels and decreases in estimated glomerular filtration rate (eGFR) have been reported during treatment with GIVLAARI. In the placebo-controlled study, 15% of patients receiving GIVLAARI experienced a renally-related adverse reaction. The median increase in creatinine at Month 3 was 0.07 mg/dL. Monitor renal function during treatment with GIVLAARI as clinically indicated.

Injection Site Reactions

Injection site reactions were reported in 25% of patients receiving GIVLAARI in the placebo-controlled trial. Symptoms included erythema, pain, pruritus, rash, discoloration, or swelling around the injection site. One (2%) patient experienced a single, transient, recall reaction of erythema at a prior injection site with a subsequent dose administration.

Blood Homocysteine Increased

Increases in blood homocysteine levels have occurred in patients receiving GIVLAARI. In the ENVISION study, during the open label extension, adverse reactions of blood homocysteine increased were reported in 15 of 93 (16%) patients treated with GIVLAARI. Measure blood homocysteine levels prior to initiating treatment and monitor for changes during treatment with GIVLAARI. In patients with elevated blood homocysteine levels, assess folate, vitamins B12 and B6. Consider treatment with a supplement containing vitamin B6 (as monotherapy or a multivitamin preparation).

Pancreatitis

Cases of acute pancreatitis, some severe, have been reported in patients receiving GIVLAARI. To ensure appropriate management, consider acute pancreatitis as a potential diagnosis in patients with signs/symptoms of acute pancreatitis. Consider interruption and/or discontinuation of GIVLAARI treatment for severe cases.

Drug Interactions

Concomitant use of GIVLAARI increases the concentration of CYP1A2 or CYP2D6 substrates, which may increase adverse reactions of these substrates. Avoid concomitant use of GIVLAARI with CYP1A2 or CYP2D6 substrates for which minimal concentration changes may lead to serious or life-threatening toxicities. If concomitant use is unavoidable, decrease the CYP1A2 or CYP2D6 substrate dosage in accordance with approved product labeling.

Adverse Reactions

The most common adverse reactions that occurred in patients receiving GIVLAARI were nausea (27%) and injection site reactions (25%).

INDICATION

GIVLAARI is indicated for the treatment of adults with acute hepatic porphyria (AHP).

For additional information about GIVLAARI, please see full Prescribing Information.