Emapalumab-lzsg

Video Alert: New Video Illustrates Mechanism of Action for Gamifant® (emapalumab-lzsg)

 

Waltham, Mass., June 3, 2019 — Sobi, an international biopharmaceutical company transforming the lives of people affected by rare diseases, announced the launch of a new video that shows the role of interferon gamma (IFNγ) in primary hemophagocytic lymphohistiocytosis (HLH) and how Gamifant® (emapalumab-lzsg) may help.

IFNγ plays a pivotal role in the pathogenesis of HLH by being hypersecreted, with consequent downstream effects of hypercytokinemia and inflammation. 1-3 Gamifant, a monoclonal antibody that binds to IFNγ, is shown to neutralize it by measuring a rapid and sustained reduction in the plasma concentrations of CXCL9, a chemokine induced by IFNγ.1 Today, the drug is the first and only FDA-approved treatment for pediatric (newborn and older) and adult patients with primary HLH with refractory, recurrent or progressive disease or intolerance to conventional HLH therapy.

Primary HLH is a rapidly progressive, often-fatal syndrome of hyperinflammation that usually occurs within the first year of life. The condition is rare, with fewer than 100 cases diagnosed each year in the U.S. 4, often by pediatric hematology-oncology specialists. Gamifant represents a new approach to helping critically ill primary HLH patients reach haematopoietic stem cell transplant.

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

 Please see full Prescribing Information at https://gamifant.com/pdf/Full-Prescribing-Information.pdf. Sobi has made the video available on the Business Wire website and the Gamifant website. More information about Gamifant is available at www.gamifant.com.
 

About primary haemophagocytic lymphohistiocytosis (HLH)
Primary haemophagocytic lymphohistiocytosis (HLH) is an ultra-rare, rapidly progressive, often-fatal syndrome of hyperinflammation in which massive hyperproduction of interferon gamma (IFNy) is thought to drive immune system hyperactivation, ultimately leading to organ failures. It is estimated that fewer than 100 cases of primary HLH are diagnosed each year in the US, but this is believed to represent under diagnosis. Diagnosis is challenging due to the variability in signs and symptoms, which may include fevers, swelling of the liver and spleen, severe low red and white blood cell counts, bleeding disorders, infections, neurological symptoms, organ dysfunction and organ failure. Primary HLH can rapidly become fatal if left untreated, with median survival of less than two months. The immediate goal of treatment is to quickly control the hyperinflammation and to prepare for haematopoietic stem-cell transplant. The current conventional treatment prior to transplant includes steroids and chemotherapy and are not specifically approved to treat primary HLH.

About Gamifant® (emapalumab-lzsg)
Emapalumab is a monoclonal antibody (mAb) that binds to and neutralizes interferon gamma (IFNy). In the US, Gamifant is indicated for paediatric (new born and older) and adult primary haemophagocytic lymphohistiocytosis (HLH) patients with refractory, recurrent or progressive disease, or intolerance to standard-of-care HLH therapy. Gamifant is the first and only medicine approved in the US for primary HLH, an ultra-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 is based on data from the pivotal phase 2/3 study (NCT01818492). Gamifant is indicated to be administered through intravenous (IV) infusion over one hour twice per week until haematopoietic stem cell transplant (HSCT). Visit www.gamifant.com for more information, including full US Prescribing Information.

Important Safety Information
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. 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.
Please see full
Prescribing Information for Gamifant.
You may also contact Sobi at medinfo.us@sobi.com or 866-773-5274.

About Sobi in North America
As the North American affiliate of international biopharmaceutical company Sobi™, our team is committed to Sobi’s vision of providing sustainable access to innovative therapies and transforming the lives of people affected by rare diseases. We bring something rare to rare diseases – a belief in the strength of focus, the power of agility and the potential of the people we are dedicated to serving. Our product portfolio includes multiple approved treatments, focused on immunology and genetics/metabolism. With North American 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 www.sobi-northamerica.com. For more information about Sobi, visit www.sobi.com.

References
1. Gamifant [prescribing information]. Stockholm, Sweden: Swedish Orphan Biovitrum AB; 2018.
2. Sepulveda F, de Saint Basile G. Hemophagocytic syndrome: primary forms and predisposing conditions. Curr Opin Immunol. 2017;49:20-26. doi:10.1016/j.coi.2017.08.004.
3. Jordan M, Hildeman D, Kappler J, Marrack P. An animal model of hemophagocytic lymphohistiocytosis (HLH): CD8+ T cells and interferon gamma are essential for the disorder. Blood. 2004;104(3):735-743. doi:10.1182/blood-2003-10-3413.
4.  Data on file. Stockholm, Sweden: Swedish Orphan Biovitrum AB.

For more information please contact:
Trista Morrison
781-810-0490
trista.morrison@sobi.com