Sobi North America, the North American affiliate of Swedish Orphan
Biovitrum AB (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.
ContactsFor details on how to
contact the Sobi Investor Relations Team, please click here. For
Sobi Media, click here.
About
emapalumab-lzsgEmapalumab-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
InformationInfectionsBefore 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 VaccinesDo 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
ReactionsInfusion-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 ReactionsIn 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 the full
Prescribing Information for Gamifant.
About Sobi North AmericaAs 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
https://www.sobi.com/usa or at www.sobi.com.
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 sobi.com, LinkedIn and YouTube.