- Prescription Drug User Fee Act (PDUFA) target
action date is March 20, 2023
- Submission based on positive results from the
Phase 3 bridging study demonstrating noninferior total IgG
reduction at day 29 with subcutaneously (SC) administered
efgartigimod compared to intravenous (IV) administration
November
22, 2022
Amsterdam, the
Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a
global immunology company committed to improving the lives of
people suffering from severe autoimmune diseases, today announced
the U.S. Food and Drug Administration (FDA) has accepted
for priority review a Biologics License Application (BLA) for SC
efgartigimod (1000mg efgartigimod-PH20) for the treatment of adult
patients with generalized myasthenia gravis (gMG). The application
has been granted a Prescription Drug User Fee Act (PDUFA) target
action date of March 20, 2023.
SC efgartigimod is co-formulated with
recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE®
drug delivery technology. ENHANZE facilitates the SC injection
delivery of biologics that are typically administered via IV
infusion.
“The FDA’s acceptance of our BLA is an exciting
step toward fulfilling our vision of delivering the broadest gMG
treatment offering that reflects the unique disease experience for
each patient as they navigate life with this debilitating disease.
We’re excited about the potential of SC efgartigimod to offer
patients multiple ways to receive treatment through various
administrations and an individualized dosing schedule,” said Keith
Woods, Chief Operating Officer of argenx. “With an established
PDUFA date, we are preparing for our second commercial product
launch and look forward to potentially bringing forth another
first-in-class option for gMG patients.”
The BLA submission is supported by data from the
Phase 3 ADAPT-SC study evaluating the noninferiority of the
pharmacodynamic (PD) effect of SC efgartigimod as compared with IV
administered VYVGART in adult patients with gMG. The majority of
enrolled patients were positive for acetylcholine receptor (AChR)
antibodies, but the trial also included patients where AChR
antibodies were not detected.
ADAPT-SC met its primary endpoint (p< 0.0001)
of total IgG reduction from baseline at day 29 demonstrating
noninferiority of SC efgartigimod to VYVGART. Patients treated with
SC efgartigimod achieved mean total IgG reduction of 66.4% from
baseline at day 29, compared to 62.2% reduction with VYVGART.
Results were consistent across the overall population, including
those with AChR antibodies and patients where AChR antibodies were
not detected. Further, 69.1% of patients treated with SC
efgartigimod were responders on the Myasthenia Gravis Activities of
Daily Living (MG-ADL) score. Responders are defined as having at
least a two-point improvement on the MG-ADL score for at least four
consecutive weeks. 65.5% of patients treated with SC efgartigimod
were responders on the Quantitative Myasthenia Gravis (QMG) score.
Responders are defined as having at least a three-point improvement
on the QMG score for at least four consecutive weeks. Minimal
symptom expression (MSE), a measure of symptom-free status, was
achieved in 37% of SC efgartigimod-treated patients after one
treatment cycle. Onset of effect was also consistent with the Phase
3 ADAPT study.
The safety profile for SC efgartigimod was
consistent with the ADAPT study. It was generally well-tolerated;
the most frequent adverse event being injection site reactions
(ISRs), commonly observed with biologics administered
subcutaneously. All ISRs were mild to moderate and resolved over
time. After completing ADAPT-SC, 95% of participants entered
ADAPT-SC+, a three-year open-label extension study evaluating the
long-term safety and tolerability of SC efgartigimod.
Phase 3 ADAPT-SC Trial
Design
The Phase 3 ADAPT-SC trial was a multicenter,
randomized, open-label, parallel-group study evaluating the
noninferiority of the pharmacodynamic (PD) effect of SC
efgartigimod (1000mg efgartigimod-PH20) as compared with VYVGART
(10mg/kg) in patients with gMG. The pharmacodynamic effect as
measured by percent change from baseline in total IgG levels at day
29, one week after the last dose of IV or SC efgartigimod, served
as the primary endpoint in the ADAPT-SC trial. The correlation
between total IgG reduction and clinical benefit in gMG was
demonstrated in a Phase 2 trial and the Phase 3 ADAPT trial, which
served as the basis for approval of VYVGART in the U.S., Japan and
Europe. Safety, clinical efficacy, immunogenicity and
pharmacokinetics (PK) were also assessed.
A total of 110 adult patients with gMG in North
America, Europe and Japan enrolled in the ADAPT-SC trial and were
treated. Inclusion criteria of the trial were the same as the Phase
3 ADAPT trial of VYVGART; enrolled patients had a confirmed gMG
diagnosis and an MG-ADL total score of at least 5 with greater than
50% of the total score attributed to non-ocular symptoms, at
screening and baseline. Patients were on a stable dose of at least
one gMG treatment prior to randomization, including
acetylcholinesterase inhibitors, corticosteroids or nonsteroidal
immunosuppressive drugs, and were required to remain on that stable
dose throughout the primary trial. Patients were eligible to enroll
in ADAPT-SC regardless of antibody status, including patients with
AChR antibodies (AChR-Ab+) and patients where AChR antibodies were
not detected.
Patients were randomized in a 1:1 ratio to
receive SC efgartigimod or IV efgartigimod for one treatment cycle
consisting of four doses at weekly intervals. The total study
duration was approximately 12 weeks, including seven weeks of
follow-up after the treatment cycle.
See the full Prescribing Information for VYVGART
in the U.S., which includes the below Important Safety Information.
For more information related to VYVGART in Japan, visit
argenx.jp.
IMPORTANT SAFETY INFORMATION FOR
VYVGART® (efgartigimod alfa-fcab) intravenous (IV) formulation
(U.S. PRESCRIBING INFORMATION)
What is VYVGART® (efgartigimod
alfa-fcab)?
VYVGART is a prescription medicine used to treat
a condition called generalized myasthenia gravis, which causes
muscles to tire and weaken easily throughout the body, in adults
who are positive for antibodies directed toward a protein called
acetylcholine receptor (anti-AChR antibody positive).
What is the most important information I
should know about VYVGART?
VYVGART may cause serious side effects,
including:
Infection. VYVGART may increase
the risk of infection. In a clinical study, the most common
infections were urinary tract and respiratory tract infections.
More patients on VYVGART vs placebo had below normal levels for
white blood cell counts, lymphocyte counts, and neutrophil counts.
The majority of infections and blood side effects were mild to
moderate in severity. Your health care provider should check you
for infections before starting treatment, during treatment, and
after treatment with VYVGART. Tell your health care provider if you
have any history of infections. Tell your health care provider
right away if you have signs or symptoms of an infection during
treatment with VYVGART such as fever, chills, frequent and/or
painful urination, cough, pain and blockage of nasal
passages/sinus, wheezing, shortness of breath, fatigue, sore
throat, excess phlegm, nasal discharge, back pain, and/or chest
pain.
Undesirable immune reactions
(hypersensitivity reactions). VYVGART can cause the immune
system to have undesirable reactions such as rashes, swelling under
the skin, and shortness of breath. In clinical studies, the
reactions were mild or moderate and occurred within 1 hour to 3
weeks of administration, and the reactions did not lead to VYVGART
discontinuation. Your health care provider should monitor you
during and after treatment and discontinue VYVGART if needed. Tell
your health care provider immediately about any undesirable
reactions.
Before taking VYVGART, tell your healthcare
provider about all of your medical conditions, including if
you:
Have a history of infection or you think you
have an infection.Have received or are scheduled to receive a
vaccine (immunization). Discuss with your health care provider
whether you need to receive age-appropriate immunizations before
initiation of a new treatment cycle with VYVGART. The use of
vaccines during VYVGART treatment has not been studied, and the
safety with live or live-attenuated vaccines is unknown.
Administration of live or live-attenuated vaccines is not
recommended during treatment with VYVGART.Are pregnant or plan to
become pregnant and are breastfeeding or plan to breastfeed.Tell
your health care provider about all the medicines you take,
including prescription and over-the-counter medicines, vitamins,
and herbal supplements.
What are the common side effects of
VYVGART?
The most common side effects of VYVGART are
respiratory tract infection, headache, and urinary tract
infection.
These are not all the possible side effects of
VYVGART. Call your doctor for medical advice about side effects.
You may report side effects to the US Food and Drug Administration
at 1-800-FDA-1088.
Please see the full Prescribing Information for
VYVGART and talk to your doctor.
About Efgartigimod
Efgartigimod is an antibody fragment designed to
reduce pathogenic immunoglobulin G (IgG) antibodies by binding to
the neonatal Fc receptor and blocking the IgG recycling process.
Efgartigimod is being investigated in several autoimmune diseases
known to be mediated by disease-causing IgG antibodies, including
neuromuscular disorders, blood disorders, and skin blistering
diseases, in both an intravenous and subcutaneous (SC) formulation.
SC efgartigimod is co-formulated with recombinant human
hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE® drug delivery
technology.
About VYVGART
VYVGART® (efgartigimod alfa-fcab) is a human
IgG1 antibody fragment that binds to the neonatal Fc receptor
(FcRn), resulting in the reduction of circulating immunoglobulin G
(IgG) autoantibodies. It is the first and only approved FcRn
blocker. VYVGART is approved in the United States and Europe for
the treatment of adults with generalized myasthenia gravis (gMG)
who are anti-acetylcholine receptor (AChR) antibody positive, and
in Japan for the treatment of adults with gMG who do not have
sufficient response to steroids or non-steroidal immunosuppressive
therapies (ISTs).
About Generalized Myasthenia
Gravis
Generalized myasthenia gravis (gMG) is a rare
and chronic autoimmune disease where IgG autoantibodies disrupt
communication between nerves and muscles, causing debilitating and
potentially life-threatening muscle weakness. Approximately 85% of
people with MG progress to gMG within 24 months1, where muscles
throughout the body may be affected. Patients with confirmed AChR
antibodies account for approximately 85% of the total gMG
population.
About argenxargenx is a global
immunology company committed to improving the lives of people
suffering from severe autoimmune diseases. Partnering with leading
academic researchers through its Immunology Innovation Program
(IIP), argenx aims to translate immunology breakthroughs into a
world-class portfolio of novel antibody-based medicines. argenx
developed and is commercializing the first-and-only approved
neonatal Fc receptor (FcRn) blocker in the U.S., Japan and the EU.
The Company is evaluating efgartigimod in multiple serious
autoimmune diseases and advancing several earlier stage
experimental medicines within its therapeutic franchises. For more
information, visit www.argenx.com and follow us
on LinkedIn, Twitter, and Instagram.
For further information, please contact:
Media:Kelsey Kirkkkirk@argenx.com
Investors:Beth
DelGiaccobdelgiacco@argenx.com
Forward Looking StatementsThe contents of this
announcement include statements that are, or may be deemed to be,
“forward-looking statements.” These forward-looking statements can
be identified by the use of forward-looking terminology, including
the terms “believes,” “hope,” “estimates,” “anticipates,”
“expects,” “intends,” “may,” “will,” or “should” and include
statements argenx makes concerning the acceptance of the Biologics
License Application to the U.S. Food and Drug Administration for
Subcutaneous (SC) Efgartigimod for Treatment of Generalized
Myasthenia Gravis, the potential commercial launch of SC
efgartigimod for treatment of generalized myasthenia gravis and the
long-term safety and tolerability of SC Efgartigimod. By their
nature, forward-looking statements involve risks and uncertainties
and readers are cautioned that any such forward-looking statements
are not guarantees of future performance. argenx’s actual results
may differ materially from those predicted by the forward-looking
statements as a result of various important factors. A further list
and description of these risks, uncertainties and other risks can
be found in argenx’s U.S. Securities and Exchange Commission (SEC)
filings and reports, including in argenx’s most recent annual
report on Form 20-F filed with the SEC as well as subsequent
filings and reports filed by argenx with the SEC. Given these
uncertainties, the reader is advised not to place any undue
reliance on such forward-looking statements. These forward-looking
statements speak only as of the date of publication of this
document. argenx undertakes no obligation publicly update or revise
the information in this press release, including any
forward-looking statements, except as may be required by law.
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