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Givlaari (givosiran)

General Description

Givlaari (givosiran) is prescribed for the treatment of acute hepatic porphyria in adults and adolescents aged 12 years and above.

Getting Givlaari (givosiran) in India

Givlaari (givosiran) is approved in the U.S., Europe, and other countries, but it is not yet commercially available in India. However, eligible patients in India may access this important therapy through the Named Patient Program (NPP).

MitoGENE helps patients obtain Givlaari (givosiran) legally and safely, in collaboration with their treating doctor. We assist with documentation, import coordination, and delivery—ensuring full compliance with Indian regulatory guidelines.

If you or a loved one needs Givlaari (givosiran),MitoGENE is here to guide you through every step of the process.

Disease Indications:Acute Hepatic Porphyria (AHP)

Manufacturer:Viela Bio

Usage:Subcutaneous

Medicine Approved by:

  • U.S. Food and Drug Administration (FDA)
  • European Medical Agency (EMA)
  •  Pharmaceuticals and Medical Devices Agency (PMDA)

Available Dosage Form & Package:

  • Single dose vials 189mg/ml

Shipping:Cold Chain Shipping. Certain medicines can be affected by heat, light, or improper handling. Cold chain shipping uses temperature-controlled packaging and transport provided by specialized medical couriers to keep these medicines stable and effective. Due to the added care and equipment involved, this method is often more expensive than standard shipping.

What is Givlaari (givosiran) used for?

Givlaari (givosiran) is a small interfering RNA (siRNA) therapy that targets aminolevulinate synthase 1 (ALAS1). It is approved for the treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older.[1,4]
It is supplied as a single-dose vial containing 189 mg/mL of givosiran.[1]

How does Givlaari work?

Acute hepatic porphyria (AHP) is a rare genetic condition caused by malfunction in the heme biosynthesis pathway, where one of the key enzymes is deficient. Heme is crucial for oxygen transport in the body and is regulated in the liver by the ALAS1 enzyme.[2]

Triggers can increase ALAS1 activity, leading to accumulation of harmful substances—aminolevulinic acid (ALA) and porphobilinogen (PBG)—which are toxic to nerve cells and result in the painful, and sometimes life-threatening, attacks seen in AHP.[2]

Givlaari works by silencing the ALAS1 gene, effectively lowering levels of ALA and PBG. This helps reduce the frequency and severity of AHP attacks and associated symptoms.[1,2]

Where is Givlaari approved?

Givlaari has received regulatory approval in several regions:

  • U.S. FDA: Approved on November 20, 2019, for adults with AHP[3]
  • European Medicines Agency (EMA): Approved on January 31, 2020, for adults and adolescents aged 12 and above[4]
  • Japan (PMDA): Approved on June 23, 2021

It has also received Priority Review, Breakthrough Therapy, and Orphan Drug designations by the FDA and EMA to support development for this rare disease.[3]

Note: Approval status may vary by country. Contact our support team for region-specific information.

How is Givlaari administered?

The recommended dosage is:[1]

  • 2.5 mg/kg actual body weight, administered once monthly via subcutaneous injection

If a dose is missed, administer it as soon as possible, and then resume the regular monthly schedule.

If liver enzyme levels (transaminases) rise significantly, the dose may be reduced to 1.25 mg/kg monthly. If liver levels stabilize, the original 2.5 mg/kg dose can be resumed.

Always consult your treating physician for personalized dosing and monitoring.

What are the possible side effects of Givlaari?

Common side effects (reported in ≥20% of patients):[1]

  • Nausea
  • Reactions at the injection site

Serious side effects include:[1]

  • Severe allergic (anaphylactic) reactions
  • Liver function abnormalities
  • Kidney issues
  • Persistent injection site reactions

Use in special populations

Givlaari may pose risks during pregnancy and breastfeeding. It is advised to avoid pregnancy and lactation while on this medication.[1]

For a complete list of potential adverse effects and precautions, refer to the official prescribing information.[1]

References

1. Full prescribing information [FDA]: Givlaari (givosiran) [PDF]
Alnylam Pharmaceuticals, Nov 20, 2019

2. Understanding Acute Hepatic Porphyria
Alnylam Pharmaceuticals, last checked on Aug 19, 2020

3. FDA approves first treatment for inherited rare disease
FDA press release, Nov 20, 2019 4. First treatment for acute hepatic porphyria Share
EMA press release, Jan 31, 2020

How to Access Givlaari (givosiran) in India in 4 Simple Steps with MitoGENE via the Named Patient Program (NPP)

If Givlaari (givosiran) is not yet approved or available commercially in India, MitoGENE can help you access it through a legal pathway. Here’s how:

    Upload Prescription

    Upload Patient Consent Form

    Patient Consent Form
    Download

    Please download and complete this form, including your signature, and attach it to the request form or email it to info@mitogene.org along with the patient details.