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We work closely with individuals impacted by rare disease, caregivers, healthcare professionals, and academic and industry partners to help ensure our products deliver value to people living with rare diseases. We apply a deliberate and careful approach to the production and commercialization of all our unique products and services.

From research to production, we are committed to making a difference and reducing the impact of rare diseases on patients and families.

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Rare Inborn Errors of Metabolism Rare Immunodeficiency Rare Hematology

Now FDA Approved!

Elfabrio is an FDA-approved PEGylated enzyme replacement therapy (ERT) indicated for the treatment of Fabry disease in adult patients.

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Indication and Important Safety Information

Indication

Elfabrio® (pegunigalsidase alfa-iwxj) is a prescription infusion medicine used to treat adults with confirmed Fabry disease.

Important Safety Information

What is the most important information I should know about Elfabrio?

Severe allergic reactions (hypersensitivity reactions), including anaphylaxis, may occur during and after Elfabrio treatment. If severe allergic reactions or anaphylaxis occurs during treatment, your healthcare provider will immediately stop the infusion and provide appropriate medical care. If these reactions should occur after treatment, seek immediate medical care.

What should I know about Elfabrio infusions?

Your healthcare provider may give you other medications prior to your Elfabrio infusions to help manage allergic reactions and infusion-related side effects. They will explain how to recognize the signs and symptoms of these allergic reactions and infusion-related side effects. If these signs and symptoms occur, it’s important for you to seek immediate medical care. If the reaction is mild to moderate, your healthcare provider may choose to slow the infusion rate or withhold the dose.

In clinical trials, 41 patients (29%) experienced an infusion-related side effect. The most common signs and symptoms of an infusion-related reaction with Elfabrio were hypersensitivity, nausea, chills, itchy skin, rash, chest pain, dizziness, vomiting, feelings of weakness, pain, sneezing, shortness of breath, nasal congestion, throat irritation, abdominal pain, skin redness, diarrhea, burning sensation, nerve pain, headache, tingling or numbness, shaking movements, agitation, increased body temperature, flushing, slow heart rate, muscle pain, high blood pressure, and low blood pressure.

Your healthcare provider will do blood and urine tests to check your kidney function during treatment with Elfabrio.

The most common side effects of Elfabrio include infusion-related side effects, common cold, headache, diarrhea, fatigue, nausea, back pain, pain in the limbs, and sinus infection.

Please see Full Prescribing Information for Elfabrio.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Now FDA Approved!

Lamzede is the first and only FDA-approved enzyme replacement therapy indicated for the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients.

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Important Safety Information

Indication

Lamzede® (velmanase alfa-tycv) is intended for the treatment of non-central nervous system symptoms of alpha-mannosidosis in adult and pediatric patients.

Important Safety Information

What is the most important information I should know about Lamzede?

Severe allergic reactions (hypersensitivity reactions) including anaphylaxis, may occur during and after Lamzede treatment. If severe allergic reactions or anaphylaxis occur during treatment, your healthcare provider will immediately stop the infusion and provide appropriate medical care. If these reactions should occur after treatment, seek immediate medical care.

What should I know about infusions?

Your healthcare provider may give you other medications prior to your next infusion to help manage allergic reactions and infusion-related side effects. They will explain how to recognize the signs and symptoms of these allergic reactions and infusion-related side effects. If these signs and symptoms occur, it’s important for you to seek immediate medical care.

What are the common signs and symptoms of an allergic reaction or infusion-related side effects with Lamzede?

In clinical trials, some patients experienced signs and symptoms of an allergic reaction, which included bluish skin discoloration, low blood pressure, vomiting, hives, skin redness, facial swelling, fever, and involuntary movements.
In clinical trials, when patients experienced an infusion-related side effect, the most common signs were fever, chills, skin redness, vomiting, cough, itching, rash, and pink eye.

Are there certain people who should or should not take Lamzede?

Lamzede may cause harm to your unborn baby. For females who are able to become pregnant, your healthcare provider should do a pregnancy test before you start treatment with Lamzede. Tell your healthcare provider right away if you become pregnant or think you may be pregnant. You should use effective birth control during treatment with Lamzede and for at least 14 days after the last dose.

What are the most common side effects of Lamzede?

Lamzede can cause side effects including severe allergic reactions and anaphylaxis, common cold, fever, headache, and joint pain or stiffness.

Please see Full Prescribing Information for Lamzede.

You are encouraged to report negative side effects of prescription drugs to the FDA.
Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Revcovi is the only FDA-approved enzyme replacement therapy indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.

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WARNINGS AND PRECAUTIONS

Injection site bleeding in patients with thrombocytopenia: Increased risk of local bleeding in patients with thrombocytopenia; should not be used if thrombocytopenia is severe.
Delay in improvement of immune function: Protect immune deficient patients from infections until improvement in immune function.

ADVERSE REACTIONS

The most commonly reported adverse reactions were cough (50%) and vomiting (33%).

In addition, the following post-marketing reports for the same class of enzyme replacement therapy used in the treatment of ADA-SCID may also be seen with Revcovi treatment:

Hematologic events: hemolytic anemia, autoimmune hemolytic anemia, thrombocythemia, thrombocytopenia, and autoimmune thrombocytopenia
Dermatological events: injection site erythema, urticaria
Lymphomas

IMPORTANT MONITORING INFORMATION

Treatment with Revcovi should be monitored by measuring trough plasma ADA activity and trough dAXP levels for maintenance of therapeutic targets. If a persistent decline in plasma ADA activity occurs, immune function and clinical status should be monitored closely, and precautions should be taken to minimize the risk of infection.

INDICATION

Revcovi® (elapegademase-lvlr) is indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.

You are encouraged to report negative side effects of prescription drugs to the FDA.
Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see Full Prescribing Information.

Ferriprox is an FDA-approved prescription medicine used to treat iron overload from blood transfusions in people with thalassemia syndromes, sickle cell disease, and other anemias.

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Important Safety Information

Indication

Ferriprox® (deferiprone) is a prescription medicine used to treat iron overload from blood transfusions in people with:

thalassemia syndromes
sickle cell disease or other anemias

Ferriprox Tablets are for adults and children ≥8 years of age; Ferriprox Oral Solution is for patients ≥3 years of age.

It is not known if Ferriprox is safe and effective to treat iron overload due to blood transfusions:

in people with myelodysplastic syndrome or Diamond Blackfan anemia
in children less than 3 years of age

What is the most important information I should know about Ferriprox?

Ferriprox can cause serious side effects, including a very low white blood cell count.

One type of white blood cell that is important for fighting infections is called a neutrophil. If your neutrophil count is low (neutropenia), you may be at risk of developing a serious infection that can lead to death. Neutropenia is common with Ferriprox and can become severe in some people. Severe neutropenia is known as agranulocytosis. If you develop agranulocytosis, you will be at risk of developing serious infections that can lead to death.

Your healthcare provider will do a blood test before you start Ferriprox and regularly during treatment to check your neutrophil count. If you develop neutropenia, your healthcare provider should check your blood counts every day until your white blood cell count improves. Your healthcare provider may temporarily stop treatment with Ferriprox if you develop neutropenia or infection.

Stop taking Ferriprox and call your healthcare provider or get medical help right away if you develop any of these symptoms of infection: fever, sore throat or mouth sores, flu-like symptoms, or chills and severe shaking.

It is important for you to have your white blood cell count checked within 24 hours of developing symptoms of an infection to see if you have severe neutropenia (agranulocytosis). Do not delay getting medical care if you are unable to reach your healthcare provider.

Do not take Ferriprox if you are allergic to deferiprone or any of the ingredients in Ferriprox.

Before you take Ferriprox, tell your healthcare provider about all of your medical conditions, including if you: have liver problems, are pregnant or plan to become pregnant. Ferriprox can harm your unborn baby. You should avoid becoming pregnant during treatment with Ferriprox. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Ferriprox. For females who are able to become pregnant, your healthcare provider should do a pregnancy test before you start treatment with Ferriprox. You should use effective birth control during treatment with Ferriprox and for at least 6 months after the last dose. For males with female partners who are able to become pregnant, you should use effective birth control during treatment with Ferriprox and for at least 3 months after the last dose. Talk to your doctor if you are breastfeeding or plan to breastfeed. It is not known if Ferriprox passes into your breast milk. Do not breastfeed during treatment with Ferriprox and for at least 2 weeks after the last dose.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Avoid drinking alcohol during treatment with Ferriprox tablets (2 times a day). This may cause a faster release of the medicine.

What are other possible side effects of Ferriprox?

Ferriprox can cause serious side effects, including increased liver enzyme levels in your blood. Your healthcare provider should do blood tests to check your liver function before you start and then monthly during treatment with Ferriprox. Your healthcare provider may temporarily stop treatment with Ferriprox if you develop increased liver enzyme levels and they continue to be increased.

Ferriprox can cause decreased levels of zinc in your blood. Your healthcare provider will do blood tests to check your zinc levels during treatment with Ferriprox and may prescribe a zinc supplement for you if your zinc levels are low.

The most common side effects of Ferriprox in people with thalassemia include nausea, vomiting, stomach-area (abdominal) pain, joint pain, abnormal liver function tests, and low white blood cells.

The most common side effects of Ferriprox in people with sickle cell disease or other anemias include fever, stomach-area (abdominal) pain, bone pain, headache, vomiting, pain in arms or legs, sickle cell anemia with crisis, back pain, abnormal liver function tests, joint pain, mouth and throat pain, common cold, low white blood cells, cough, and nausea.

Ferriprox may cause a change in urine color to reddish-brown. This is not harmful and is expected during treatment with Ferriprox.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see Full Prescribing Information, including boxed WARNING, and Medication Guide.

For the updated versions of the Summary of Product Characteristics (SmPC) and the Patient Information Leaflet (PIL), please refer to the EMA (European Medicines Agency) website.

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In accordance with the current legislation, we are unable to provide any information about our drugs concerning health issues. Anyone requiring these details should contact their treating physician.

For the updated versions of the Summary of Product Characteristics (SmPC) and the Patient Information Leaflet (PIL), please refer to the EMA (European Medicines Agency) website.

Visit website

In accordance with the current legislation, we are unable to provide any information about our drugs concerning health issues. Anyone requiring these details should contact their treating physician.

For the updated versions of the Summary of Product Characteristics (SmPC) and the Patient Information Leaflet (PIL), please refer to the EMA (European Medicines Agency) website.

Visit website

In accordance with the current legislation, we are unable to provide any information about our drugs concerning health issues. Anyone requiring these details should contact their treating physician.

For the updated versions of the Summary of Product Characteristics (SmPC) and the Patient Information Leaflet (PIL), please refer to the EMA (European Medicines Agency) website.

Visit website

In accordance with the current legislation, we are unable to provide any information about our drugs concerning health issues. Anyone requiring these details should contact their treating physician.