Sobi Receives US FDA Fast Track Designation for Emapalumab-lzsg being investigated in Macrophage Activation Syndrome

WALTHAM, Mass., May 24, 2024 (GLOBE NEWSWIRE) -- Sobi North America, the North American affiliate of (Sobi^®), today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to emapalumab-lzsg being investigated as a potential therapeutic option in patients with Macrophage Activation Syndrome (MAS). Emapalumab is a fully human, anti-IFNγ monoclonal antibody that binds free and receptor-bound IFNγ, neutralizing its biological activity. Emapalumab is currently in Phase lll development for MAS.

Fast track designation is designed to facilitate the development and expedite the review of medicines to treat serious conditions that may fill an unmet medical need.

MAS is a severe complication of rheumatic diseases, most frequently in Still’s disease including systemic juvenile idiopathic arthritis (sJIA). Interferon gamma (INFy) is a key cytokine in the development of MAS. MAS is classified as a form of haemophagocytic lymphohistiocytosis (HLH) and characterized by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinaemia, possibly rapidly progressing to multiple organ failure and death.

Emapalumab-lzsg is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation that can rapidly become fatal unless diagnosed and treated.

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About emapalumab-lzsg

Emapalumab-lzsg is a fully human, anti-IFNγ monoclonal antibody that binds free and receptor-bound IFNγ, neutralizing its biological activity. In the US, emapalumab-lzsg is indicated for pediatric (newborn and older) and adult primary hemophagocytic lymphohistiocytosis (HLH) patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy. Emapalumab-lzsg is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval in 2018 was based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Emapalumab is indicated for administration through intravenous infusion over one hour twice per week until hematopoietic stem cell transplantation (HSCT). The efficacy and safety of emapalumab are currently being evaluated in a phase 3 study in patients with MAS in Still’s disease or systemic lupus erythematosus (SLE) (EMERALD; NCT05001737).

U.S. Indication for Gamifant^® (emapalumab-lzsg)

Gamifant^® (emapalumab-lzsg) is an interferon gamma (IFNγ)–blocking antibody indicated for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy.

Important Safety Information

Infections

Before initiating Gamifant, patients should be evaluated for infection, including latent tuberculosis (TB). Prophylaxis for TB should be administered to patients who are at risk for TB or known to have a positive purified protein derivative (PPD) test result or positive IFNγ release assay.

During Gamifant treatment, patients should be monitored for TB, adenovirus, Epstein-Barr virus (EBV), and cytomegalovirus (CMV) every 2 weeks and as clinically indicated.

Patients should be administered prophylaxis for herpes zoster, Pneumocystis jirovecii, and fungal infections prior to Gamifant administration.

Increased Risk of Infection With Use of Live Vaccines

Do not administer live or live attenuated vaccines to patients receiving Gamifant and for at least 4 weeks after the last dose of Gamifant. The safety of immunization with live vaccines during or following Gamifant therapy has not been studied.

Infusion-Related Reactions

Infusion-related reactions, including drug eruption, pyrexia, rash, erythema, and hyperhidrosis, were reported with Gamifant treatment in 27% of patients. In one-third of these patients, the infusion-related reaction occurred during the first infusion.

Adverse Reactions

In the pivotal trial, the most commonly reported adverse reactions (≥10%) for Gamifant included infection (56%), hypertension (41%), infusion-related reactions (27%), pyrexia (24%), hypokalemia (15%), constipation (15%), rash (12%), abdominal pain (12%), CMV infection (12%), diarrhea (12%), lymphocytosis (12%), cough (12%), irritability (12%), tachycardia (12%), and tachypnea (12%).

Additional selected adverse reactions (all grades) that were reported in less than 10% of patients treated with Gamifant included vomiting, acute kidney injury, asthenia, bradycardia, dyspnea, gastrointestinal hemorrhage, epistaxis, and peripheral edema.

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About Sobi North America

As the North American affiliate of international biopharmaceutical company Sobi, the Sobi North America team is committed to Sobi’s vision of being a leader in providing innovative treatments that transform lives for individuals with rare diseases. Our product portfolio includes multiple approved treatments focused on immunology, hematology and specialty care. With U.S. headquarters in the Boston area, Canadian headquarters in the Toronto area, and field sales, medical and market access representatives spanning North America, our growing team has a proven track record of commercial excellence. More information is available at or at .

About Sobi^®

Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of hematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at , and .