Patient Experience

Help your patients rediscover their everyday

Acute hepatic porphyria (AHP) causes debilitating, potentially life-threatening attacks that can disrupt and overwhelm the lives of patients with the disease. GIVLAARI® (givosiran) may help reduce AHP attacks in adults.1,2

Mike, Lina, and Amalia are three real patients diagnosed with AHP and 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.

Real  GIVLAARI® (givosiran) patient Mike's Journey with AIP
Real GIVLAARI® patient Mike's Journey with AIP

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.

Real GIVLAARI® patient Lina's Journey with AIP
+SEE LINA’S JOURNEY
Real GIVLAARI  (givosiran) patient Amalia's Journey with AIP +SEE AMALIA'S JOURNEY
See Lina's Journey
Real GIVLAARI  (givosiran) patient Amalia's Journey with AIP

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

Life with AHP Today

Treatment

  • GIVLAARI – Once-monthly subcutaneous injection2

Day-to-day living with AIP

  • Experienced a reduction in attacks
  • Monitors/avoids common attack triggers (alcohol, smoking, certain medications, etc.)3,4
  • Has occasional fatigue while traveling

"Now that I'm experiencing fewer attacks, I'm not afraid to go out." –Mike

Real  GIVLAARI® (givosiran) patient Lina's Journey with AIP
Real GIVLAARI® patient Lina's Journey with AIP

Lina’s Journey

"The first thing I think when I hear porphyria is, debilitating."

Lina always wanted to follow in the footsteps of her parents and become a doctor. During the first year of her undergraduate program, she was diagnosed with acute intermittent porphyria (AIP) and feared she would never reach her goals.

Real GIVLAARI® patient Mike's Journey with AIP
+SEE MIKE'S JOURNEY
Real GIVLAARI  (givosiran) patient Amalia's Journey with AIP +SEE AMALIA'S JOURNEY
See Mike's Journey
Real GIVLAARI  (givosiran) patient Amalia's Journey with AIP

History with AHP

Pre-diagnosis

  • Experienced chronic nausea and headaches in high school
  • Was prescribed medication not recommended for people with AHP
  • Experienced first major attack during freshman year of college
  • After multiple tests failed to produce a diagnosis, her pain was attributed to anxiety

Post-diagnosis

  • Diagnosed after experiencing a seizure less than a year after her first attack
  • Avoided going to the hospital until symptoms were incapacitating 
  • Hospitalized every 3-4 months
  • OTC pain medications
  • Hot water/heat therapy to manage pain

Life with AHP Today

Treatment

  • GIVLAARI – Once-monthly subcutaneous injection2

Day-to-day living with AIP

  • Experienced a reduction in attacks
  • Continues to monitor/avoid common attack triggers (alcohol, smoking, certain medications, etc.)3,4

"I do still have a debilitating condition. But because I'm experiencing fewer attacks, I feel more like a healthy person." —Lina

Amalia's Journey
Amalia's Journey

Amalia's Journey

"Your pain is real; it is valid, and you don’t have to go through it alone."

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.

Real GIVLAARI® patient Lina's Journey with AIP
+SEE LINA’S JOURNEY
Mike's Journey +SEE MIKE'S JOURNEY
See Lina's Journey
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 injection2

Day-to-day living with AIP

  • Experienced a reduction in attacks
  • Monitors/avoids common attack triggers (alcohol, smoking, certain medications, etc.)3,4
  • Has occasional fatigue and nausea during menstrual cycle

"Porphyria is part of me. It has given me a passion for helping others heal." –Amalia

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.

Connect your patients with Alnylam Assist® for personalized support

The Alnylam Assist® team will help your patients through treatment with GIVLAARI:

Case managers icon

Alnylam Case Managers

Case Managers are available to help your patients navigate insurance benefits, get started on GIVLAARI, and provide ongoing support.

Patient Education Liaisons (PELs)

Alnylam Patient Education Liaisons (PELs)

Alnylam PELs have backgrounds in nursing and are experienced educating about acute hepatic porphyria. PELs are employees of Alnylam Pharmaceuticals and do not provide medical advice.

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

Alnylam Assist® Brochure – Helps patients get access to GIVLAARI with customized support

Alnylam Assist® Contact Card Magnet – A quick reference for patients to contact their dedicated Alnylam Case Manager

Journal & Pen – For patients to track their treatment journey

Getting started with  GIVLAARI® (givosiran) brochure

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.

Do you have more questions about GIVLAARI?

Schedule an informative discussion with a GIVLAARI Representative.

Contact a Representative

References: 1. Anderson KE, Bloomer JR, Bonkovsky HL, et al. Ann Intern Med. 2005.142:439-451. 2. GIVLAARI [prescribing information]. Cambridge, MA: Alnylam Pharmaceuticals, Inc. 3. Balwani M, Wang B, Anderson KE, et al; Porphyrias Consortium of the Rare Diseases Clinical Research Network. Hepatology. 2017;66:1314-1322. 4. Lin CS, Lee M, Park SB, et al. 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).

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.