argenx Announces FDA Acceptance of BLA Filing for Efgartigimod for the Treatment of Generalized Myasthenia Gravis
02 Março 2021 - 4:00AM
Regulated Information/Inside
Information
argenx Announces FDA Acceptance of BLA
Filing for Efgartigimod for the Treatment of Generalized Myasthenia
Gravis
- If approved, efgartigimod will be the first-and-only approval
of an FcRn antagonist
- Prescription Drug User Fee Act (PDUFA) target action date is
December 17, 2021
- Pre-approval access program opened in U.S. for efgartigimod for
eligible people living with gMG
Breda, the Netherlands – March 2, 2021 – argenx
(Euronext & Nasdaq: ARGX), a global immunology company
committed to improving the lives of people suffering from severe
autoimmune diseases and cancers, today announced that the U.S. Food
and Drug Administration (FDA) has accepted for review the Biologics
License Application (BLA) for intravenous (IV) efgartigimod, the
company’s investigational FcRn antagonist and lead product
candidate, for the treatment of generalized myasthenia gravis
(gMG). The FDA has set a standard 10-month review process with a
PDUFA target action date of December 17, 2021.
“This is an important milestone for argenx in our transition to
a commercial-stage company and brings us closer to our mission to
reach patients living with gMG, a debilitating neuromuscular
disease,” said Tim Van Hauwermeiren, Chief Executive Officer of
argenx. “We look forward to closely collaborating with the FDA
through the BLA review process and to potentially making our first
medicine available.”
The BLA included results from the pivotal Phase 3 ADAPT trial
evaluating the safety and efficacy of efgartigimod for the
treatment of patients with gMG. ADAPT met its primary endpoint
defined as percentage of responders on the Myasthenia Gravis
Activities of Daily Living (MG-ADL) score among acetylcholine
receptor-antibody positive (AChR-Ab+) gMG patients. 67.7% of
AChR-Ab+ patients treated with efgartigimod achieved the primary
endpoint compared with 29.7% on placebo (p<0.0001). Further, 40%
of patients treated with efgartigimod achieved minimal symptom
expression defined as MG-ADL scores of 0 (symptom free) or 1,
compared to 11.1% of those who received placebo. The safety profile
of efgartigimod was comparable to placebo. After completing ADAPT,
90% of participants entered ADAPT+, a three-year open-label
extension study evaluating the long-term safety and tolerability of
efgartigimod. There are currently 118 patients still enrolled in
ADAPT+.
argenx also announced today the opening of its pre-approval
access (PAA) program in the U.S., which will allow eligible people
living with gMG to receive treatment with efgartigimod. The purpose
of the PAA is to open availability of an investigational treatment
to people who have a high degree of unmet clinical need with gMG
and are not able to participate in a clinical trial.
argenx is also on track to submit an application for
efgartigimod to Japan’s Pharmaceuticals and Medical Devices Agency
(PMDA) in the first half of 2021 and the European Medicines Agency
(EMA) in the second half of 2021.
About Efgartigimod
Efgartigimod is an investigational antibody fragment designed to
reduce disease-causing immunoglobulin G (IgG) antibodies and block
the IgG recycling process. Efgartigimod binds to the neonatal Fc
receptor (FcRn), which is widely expressed throughout the body and
plays a central role in rescuing IgG antibodies from degradation.
Blocking FcRn reduces IgG antibody levels representing a logical
potential therapeutic approach for several autoimmune diseases
known to be driven by disease-causing IgG antibodies, including:
myasthenia gravis (MG), a chronic disease that causes muscle
weakness; pemphigus vulgaris (PV), a chronic disease characterized
by severe blistering of the skin; immune thrombocytopenia (ITP), a
chronic bruising and bleeding disease; and chronic inflammatory
demyelinating polyneuropathy (CIDP), a neurological disease leading
to impaired motor function.
About Myasthenia Gravis
Myasthenia gravis (MG) is a rare and chronic autoimmune disease,
often causing debilitating and potentially life-threatening muscle
weakness. More than 85% of people with MG progress to generalized
MG (gMG) within 18 months, where muscles throughout the body may be
affected, resulting in extreme fatigue and difficulties with facial
expression, speech, swallowing and mobility. In more
life-threatening cases, MG can affect the muscles responsible for
breathing. There are approximately 65,000 people in the United
States and 20,000 people in Japan living with the disease
About argenx
argenx is a global immunology company committed to improving the
lives of people suffering from severe autoimmune diseases and
cancer. 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 is evaluating efgartigimod in
multiple serious autoimmune diseases, and cusatuzumab in
hematological cancers in collaboration with Janssen. argenx is also
advancing several earlier stage experimental medicines within its
therapeutic franchises. argenx has offices in Belgium, the United
States, and Japan. For more information, visit www.argenx.com and
follow us on LinkedIn at https://www.linkedin.com/company/argenx/
and Twitter at https://twitter.com/argenxglobal.
Media:Kelsey Kirkkkirk@argenx.com
Investors:Beth
DelGiaccobdelgiacco@argenx.com
Joke Comijn (EU)jcomijn@argenx.com
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or may be deemed to be, forward-looking statements. These
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including in argenx’s most recent annual report on Form 20-F filed
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