We understand that a diagnosis of primary HLH can be overwhelming, but we hope you can take some comfort in the fact that treatment options are available, and that Gamifant was specifically designed to treat primary HLH.

By working with your healthcare team and learning all you can about Gamifant, you and your loved ones can be prepared for the journey ahead.

How Gamifant works

Gamifant is the first and only FDA-approved treatment made specifically for people with primary HLH who have refractory, recurrent, or progressive disease, or intolerance to conventional therapy.

If your doctor decides that Gamifant is right for you or your child, learning all you can will help you prepare for treatment.

Gamifant targets the main cause of primary HLH

There are many different cytokines involved in primary HLH. These may include interleukin-1 (IL-1), interleukin-6 (IL-6), and tumor-necrosis factor alpha (TNF-α). However, one cytokine called interferon gamma (IFNγ) plays an important role.

IFNy connects to macrophages, directly telling them to begin the process of making more cytokines. This continued production of cytokines contributes to the symptoms and inflammation that can happen with primary HLH.

Gamifant was specifically developed to stop IFNγ from connecting to the macrophage’s receptors by connecting to it first. By blocking IFNγ activity, Gamifant helps manage inflammation associated with primary HLH symptoms.

Gamifant was specifically developed to stop IFNγ from connecting to the macrophage’s receptors by connecting to it first. By blocking IFNγ activity, Gamifant helps manage inflammation associated with primary HLH symptoms.

About Gamifant

Gamifant was proven to help treat primary HLH safely and effectively

Gamifant was proven to be safe and effective for most people in a clinical trial and was approved by the FDA in 2018. In the trial, investigators looked at:

  • How effective Gamifant was for people with primary HLH who had received conventional therapy. These people either hadn’t responded to or had been unable to tolerate conventional primary HLH therapy, or their disease came back
  • How safe Gamifant was for these people, plus several more who had not received conventional treatment

Talk to your doctor about whether Gamifant might be right for you or your child.

How Gamifant could help

For those who did not respond to conventional therapy, couldn’t tolerate it, whose symptoms came back or did not improve, or who experienced rapidly progressive disease, Gamifant helped:

63% of people had positive improvements in the signs and symptoms of primary HLH

70% of people with primary HLH proceeded to transplant

How clinical trials work

A clinical trial is a program run by researchers (or investigators) to evaluate a new medical treatment. The investigators look at how well a treatment works, and how safe it may be for patients.

  • Clinical trials happen in phases. If a treatment is shown to be safe and effective in the first, it moves on to the next. To be considered for approval by the FDA, the treatment must move through 3 phases
  • After 3 phases, the FDA will look at all the information from the trial and decide whether or not the benefits of the treatment outweigh the potential risks before approving it for use

Talk to your doctor about whether Gamifant might be right for you or your child.

Potential side effects

When considering Gamifant, it is important to discuss any potential side effects with your doctor.

Risk of serious infections

Because Gamifant affects the immune system, it may lower a patient’s ability to fight infections. Gamifant may increase the risk of serious infections, including:

  • Tuberculosis (TB)
  • Histoplasmosis
  • Herpes zoster infection (chicken pox and shingles)
  • Other infections caused by viruses, fungi, or bacteria that can spread throughout the body

Doctors will test people for TB before starting Gamifant, and they may prescribe a treatment for TB if a person is at risk or has a positive TB test. Some people may also be considered for therapies that can help prevent certain infections before they receive Gamifant.

Other potential side effects

There are some serious side effects that can happen during treatment with Gamifant, including fever, high blood pressure (hypertension), and infusion reactions.*

*Infusion reactions such as rash and fever are common with Gamifant and usually happen during or shortly after treatment. Your doctor may temporarily stop the infusion and treat symptoms before continuing therapy if you or your child experiences severe reactions.

These are not all of the possible side effects of Gamifant. Talk to your doctor for medical advice about side effects. In addition, you may report side effects to the FDA by calling 1-800-FDA-1088.

Dosing

If your doctor prescribes Gamifant, you or your child will most likely receive treatment in the hospital.

Gamifant is given by intravenous (IV, or in a vein) infusion. The infusion will take about an hour. Your doctor or infusion nurse will watch for side effects during the infusion.

Gamifant is given 2 times a week (every 3 to 4 days) until you or your child begins conditioning for transplant or no longer needs treatment.

Gamifant is used with a steroid called dexamethasone, which is often given on a daily schedule. Over time, your doctor may reduce the amount of dexamethasone.

Dosing may change throughout treatment

Your doctor will start by giving a low dose of Gamifant at first. Depending on how you or your child responds to that dose, the amount may be increased.

This amount may change over the course of treatment depending on how you or your child responds to the dose.

Additional help is available for you and your family

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