Rare Diseases

The commitment of Neopharmed Gentili in the rare disease field is aimed at improving the lives of people living with rare conditions such as hereditary angioedema (HAE).

Our journey is guided by what truly matters to those living with a rare disease. Where every improvement can change a life.

We combine deep expertise in rare diseases with a strong commitment to patient-centered innovation. We are driven by curiosity. Guided by empathy. And always standing alongside the rare disease community.

We provide first-in-class or best-in-class oral therapies based on small molecules and proteins for people living with HAE and other rare diseases. We turn scientific promise into results that patients can experience every day.

Together, we are connected to the rare, committed to life.

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Our Pipeline

We are developing a product portfolio focused on rare diseases, with the ambition to provide innovative, high-value therapies in areas characterized by significant unmet medical need. Our strategy combines long-term development, established expertise in the field of hereditary angioedema (HAE), and a scalable European platform to support patients, healthcare professionals, and healthcare systems.

The expansion of our pipeline strengthens our position in the rare disease sector, already well-established in hereditary angioedema (HAE), and reflects our ambition to become a leading player in Europe.

Navenibart

Navenibart is a long-acting plasma kallikrein inhibitor currently being evaluated in a Phase 3 clinical program, which is expected to enable the submission of a potential marketing authorization application by the end of 2027.

Through an exclusive agreement with BioCryst Pharmaceuticals, Neopharmed Gentili (through its Irish subsidiary) has acquired the rights to commercialize Navenibart in Europe, consolidating its position as a leading platform in Europe in the rare disease sector and strengthening a scalable ecosystem capable of supporting both marketed products and late-stage pipeline assets.

HCP

Organized by therapeutic areas, in this section, accredited physicians can download all informational materials and specific updates related to each medicine distributed by Neopharmed Gentili.

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SUSTAINABILITY

The planet’s ecological balance is in danger. We can’t sit back and watch.

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