#United States #Stockholm #Sobi #Gamifant #HLH

FDA Grants Priority Review for Sobi's Gamifant®

In a significant development for patients suffering from severe forms of Still's disease, the U.S. Food and Drug Administration (FDA) has announced a priority review for Swedish biotech company Sobi's supplemental Biologics License Application (sBLA) for Gamifant® (emapalumab-lzsg). This therapy is aimed at treating adult and pediatric patients experiencing hemophagocytic lymphohistiocytosis (HLH) and macrophage activation syndrome (MAS), particularly in cases unresponsive to glucocorticoids or where such treatments are contraindicated. The PDUFA date for this application is set for June 27, 2025, providing hope for many in desperate need of effective therapies.

HLH/MAS represents a severe inflammatory response that can arise from rheumatic diseases such as systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease (AOSD). Patients with HLH/MAS experience dangerous hyperinflammation, which can lead to high persistent fevers, excessive ferritin levels, and dysfunction in multiple organ systems. Dr. Lydia Abad-Franch, Sobi's Head of Research & Development and Chief Medical Officer, emphasizes that there are currently no approved therapies available for managing these life-threatening complications.

Sobi’s Gamifant® acts by specifically neutralizing interferon gamma (IFN-γ), a crucial component that drives hyperinflammation in HLH. The potential approval of this drug could significantly reduce the reliance on high-dose glucocorticoids, which can carry a myriad of adverse effects.

The priority review stems from promising data compiled from two pivotal studies: the EMERALD (NCT05001737) and NI-0501-06 (NCT03311854). These trials collectively enrolled 39 patients, revealing that 53% had achieved a complete response by week eight, while a remarkable 85% had responded positively at some point during the studies. Furthermore, during the first two weeks of treatment, the average dosage of glucocorticoids administered to participants dropped by an impressive 70.1%, showcasing the potential for improved management of this complex condition with Gamifant®.

Initially approved in 2018, Gamifant® had already made a name for itself as the first FDA-approved treatment for primary HLH across different age demographics, particularly those who face refractory or recurrent forms of the disease. It’s crucial to note that primary HLH is notably rare, manifesting primarily within the first year of life and can quickly become fatal if left untreated. The service method involves administering Gamifant® intravenously over the course of an hour, twice weekly until a hematopoietic stem cell transplant can be performed.

Beyond merely addressing HLH/MAS, Gamifant® signifies an evolution in treatment paradigms for severe rheumatic conditions linked to abnormal immune responses. As Sobi continues to push the boundaries of therapeutic intervention, the expectations surrounding Gamifant® are high, with its effectiveness potentially reshaping patient management strategies.

As Sobi looks ahead, the company remains steadfast in its commitment to addressing the needs of patients afflicted by rare and complex diseases. With approximately 1,800 dedicated employees and a broad international presence, Sobi's focus is on providing consistent access to innovative medicines, exemplified in their ongoing work in hematology, immunology, and specialized care. Sobi's future endeavors and the outcomes of the Gamifant® priority review will be crucial for both the company and the larger patient community facing these challenging health conditions.