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

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.

This press release features multimedia. View the full release here:
https://www.businesswire.com/news/home/20190603005072/en/

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 [email protected]
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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20190603005072/en/