Chiesi Global Rare Diseases Demonstrates Its Ongoing Commitment To The Rare Disease Community At The Society Of Inborn Errors Of Metabolism (SSIEM) 2026 Annual Symposium
| 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 or call 1-800-FDA-1088.
Important Safety Information and 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 or call 1-800-FDA-1088.
About Fabry Disease
Fabry disease is a rare, inherited lysosomal storage disorder caused by mutations in the GLA gene, which leads to a deficiency of the enzyme alpha-galactosidase A. This deficiency results in an accumulation of a fatty substance called globotriaosylceramide (GL-3) in the body's cells, affecting the heart, kidneys, skin, nervous system, and other organs. Fabry disease can cause a range of serious signs and symptoms, including fatigue, chronic pain, gastrointestinal issues, decreased ability to sweat, progressive kidney failure, heart complications, and increased risk of stroke.
The condition affects both males and females and can present from childhood through adulthood, often with delayed diagnosis or misdiagnosis. While Fabry disease is rare, early detection and access to appropriate treatment - such as enzyme replacement therapy or pharmacological chaperone therapy - are critical in managing symptoms and slowing disease progression.
About Alpha-mannosidosis
Alpha-mannosidosis is an ultra-rare, inherited lysosomal storage disorder caused by mutations in the MAN2B1 gene, which results in a deficiency of the enzyme alpha-mannosidase. This deficiency leads to the accumulation of oligosaccharides, within the body's cells, causing progressive damage to multiple organs and tissues. Alpha-mannosidosis can affect the musculoskeletal system, hearing, immune system, nervous system, and other organs, and is associated with a wide range of signs and symptoms, including skeletal abnormalities, impaired mobility, hearing loss, cognitive impairment, immune dysfunction, and behavioral or mental health challenges.
The condition affects both children and adults and may present with few or mild symptoms early in life, progressing over time as oligosaccharides accumulate. While alpha-mannosidosis is rare, timely diagnosis and appropriate disease management are important to help address symptoms and support long-term outcomes for affected individuals.
About Chiesi Group
Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The Company's mission is to improve people's quality of life and act responsibly towards both the community and the environment.
By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi's commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The Company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035.
With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group's research and development center in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden.
For more information visit .
About Chiesi Global Rare Diseases
Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system.
For more information visit .
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Chiesi Global Rare Diseases Media Contact
Sky Striar
LifeSci Communications
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