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Sanofi's efdoralprin alfa earns orphan designation in the EU for
alpha-1 antitrypsin deficiency related emphysema
Paris, December 17, 2025. The European Medicines Agency (EMA) has granted orphan designation to efdoralprin alfa (SAR447537,
formerly known as INBRX-101), an investigational recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, for the potential
treatment of alpha-1 antitrypsin deficiency (AATD) related emphysema, a rare respiratory condition with great unmet medical need.
The EMA grants orphan designation to potential new medicines addressing rare, life-threatening or debilitating medical diseases or conditions that
affect no more than 5 in 10,000 individuals in the EU.
demonstrated superiority to a standard of care plasma-derived therapy in adults with AATD when dosed every three weeks (Q3W) or every
four weeks (Q4W), meeting all primary and key secondary endpoints in the global phase 2 ElevAATe study (clinical study identifier: NCT05856331).
The US Food and Drug Administration (FDA) previously granted both fast track and orphan drug designation to
efdoralprin alfa for the treatment of AATD related emphysema. Efdoralprin alfa is currently in clinical development, and its safety and efficacy have not been evaluated by any regulatory authority. Sanofi plans to present the data at a
forthcoming medical meeting and engage with global regulatory authorities on the appropriate next steps.
About efdoralprin alfa
Efdoralprin alfa (SAR447537, formerly known as INBRX-101) is a recombinant human
AAT-Fc fusion protein being investigated in adults with AATD emphysema, with Q3W or Q4W dosing. The investigational restorative recombinant treatment is being studied to restore functional AAT levels to the
normal range and inhibit neutrophil elastase, an enzyme that can cause lung tissue damage in patients with AATD. Efdoralprin alfa was granted fast track designation and orphan drug designation by the FDA for the treatment of AATD emphysema in
addition to this latest orphan designation from the EMA.
AATD is a rare, inherited disorder characterized by low levels or absence of AAT, a protein produced by the liver that protects the lungs from
inflammation and damage. The disease causes progressive deterioration of the tissue of the lungs and liver. Without adequate AAT levels,
affected individuals often experience lung damage and develop COPD, including emphysema, and in severe forms of the disease, patients can sometimes require lung transplantation. Plasma-derived therapies were introduced in 1987 to treat the condition
but since then, no new therapies have been introduced. About 235,000 people worldwide live with AATD, with nearly 100,000 people in the US, but about 90% of individuals with AATD are likely undiagnosed.
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Evan Berland | +1 215 432 0234 | evan.berland@sanofi.com
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