Chiesi Farmaceutici

The goal of this observational registry is to evaluate the safety and outcomes of pregnancy and lactation in women with Fabry disease who are exposed to pegunigalsidase alfa within 30 days prior to conception and/or during pregnancy and lactation. The main objectives are to: * Assess pregnancy outcomes, including maternal and infant health. * Evaluate the occurrence of congenital malformations and other neonatal outcomes. This is a global, decentralized, single-arm, prospective and retrospective registry planned to enroll participants over a 10-year period. Eligible patients may be enrolled by their physician or may self-enroll, where permitted by local regulations. Data will be collected through a secure web-based platform, allowing patients and physicians to enter information via electronic case report forms (eCRFs). Pregnancy and clinical outcomes will be documented throughout pregnancy and up to 12 months post-birth. Data from self-enrolled patients will be confirmed by their primary care or attending physician. This registry is observational and does not impact clinical care or treatment decisions.Show more

The purpose of this study is to evaluate the efficacy, safety, and tolerability at Week 24 of 2 doses of CHF10067 (zampilimab) in participants with idiopathic pulmonary fibrosis (IPF). It is a phase IIb, multicentre, randomised, double-blind, placebo-controlled, three-arm parallel-group study. A total of 240 participants with IPF (Idiomatic Pulmonary Fibrosis) will be randomised in approximately 150 investigational sites in North and Latin America, Europe, Asia, and Oceania.Show more

In patients with epidermolysis bullosa (EB), collagen does not form properly, so their skin is very fragile and blisters easily. Such patients are also at greatly increased risk of developing skin cancers. Filsuvez is a topical gel used to promote healing of skin lesions in patients with certain types of EB. In this observational study, patients with either dystrophic EB (DEB) or junctional EB (JEB) will receive standard of care treatment, whether Filsuvez or something else, and will be followed for a minimum of 3 years. The main purpose is to see if the use of Filsuvez affects the likelihood of developing skin malignancies in these patient populations.Show more

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.

PEGASO is an observational study designed to collect prospective data on the effectiveness and safety of pegunigalsidase alfa in adult patients with Fabry disease, being treated or planning to start treatment, under real-world setting.

The goal of this clinical trial is to learn if an exercise programme started shortly after diagnosis improves the fitness and mental wellbeing of patients with pulmonary hypertenion over and above medication alone. It will also learn about whether extra support can help patients keep up with exercise in the long term. The main questions it aims to answer are: * Doe exercise improve how far patients can walk over six minutes * Does exercise improve quality of life scores * Does exercise improve mental health scores * Does extra support after an exercise programme help patients to continue exercising in the long term Researchers will compare the exercise programme to a control group (a group of similar patients who do not receive the exercise programme) to see the effect that exercise has. Participants in the exercise programme group will: * Undergo an exercise programme for 12 weeks, starting around 3 months after their diagnosis * They will undertake the exercise programme at home, remotely supervised by the research team with regular contact * Visit the clinic at the end of the programme for checkups and tests * Keep a diary of their exercise and how they are feeling Participants in the control group will: -Still undergo the exercise programme, but this will happen at a delayed time, starting around 6 months after diagnosis, to allow for a comparison between the two groups. Following completion of the exerise programme: * Half of participants will receive extra support to help them continue to exercise * The other half will not receive any additional support * All patients will be reviewed 1 year following the completion of their exercise programme to monitor their ongoing exercise levels.Show more

The aim of this study is to evaluate the safety and efficacy of pegunigalsidase alfa in Japanese patients (adults and adolescents) affected by Fabry disease. It is planned of a total of approximately 16 male and female Fabry disease patients between the ages of 13 and 70 years to be part of the study. The study is conducted in Japan.Show more

The goal of this clinical trial is to learn if Oleogel-S10 gel works to treat skin wounds from two types of inherited epidermolysis bullosa (EB): junctional EB (JEB) or dystrophic EB (DEB) in the Japanese population. Children and adults may participate. The trial also looks at the safety of Oleogel-S10 gel. The main questions it will answer are: * Does Oleogel-S10 gel close wounds from JEB or DEB within 45 days or reduce the size of the wounds? * Are there any medical problems when using Oleogel-S10 gel? * How much of the drug ends up in your blood? The study has 2 parts. In Part 1, participants will: * Apply Oleogel-S10 gel to the EB wound dressings at least once every 4 days for 45 days. * Visit the clinic once every 2 weeks for checkups and tests. If participants complete Part 1, they may participate in Part 2 if they choose to. In Part 2, participants will continue to receive Oleogel-S10 gel until the product is available to buy in Japan or until the trial is stopped by the company.Show more

Chiesi Farmaceutici has 7 FDA-approved drugs with a rare disease use that we track, as of November 2026.

Filsuvez (birch triterpenes), approved 2023: Epidermolysis Bullosa.

Ferriprox (deferiprone): Thalassemia (since 2011), Sickle Cell Disease (since 2021).

Mycapssa (octreotide (oral capsules)), approved 2020: Acromegaly.

BETHKIS (tobramycin), approved 2012: Cystic Fibrosis.

ELFABRIO (pegunigalsidase alfa), approved 2023: Fabry Disease.

Juxtapid (lomitapide mesylate), approved 2012: Homozygous Familial Hypercholesterolemia.

Revcovi (elapegademase-lvlr), approved 2018: Severe Combined Immunodeficiency.

Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.

Yes, for 3 of its FDA-approved rare disease medicines that we track, each checked on Chiesi Farmaceutici's own website.

Filsuvez: Chiesi Total Care, 1-800-784-5840. Offers copay help with private insurance and insurance help. Checked November 2, 2026.

Ferriprox: Chiesi Total Care, 1-866-758-7071. Offers copay help with private insurance and insurance help. Checked September 24, 2026.

Mycapssa: Chiesi Total Care, 1-833-346-2277. Offers copay help with private insurance and insurance help. Checked November 3, 2026.

Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.

Eligibility is decided by each program, so call the number listed before a prescription is filled.