Prescription Drug User Fee Act (PDUFA) target
action date is June 21, 2024
If approved, VYVGART® Hytrulo will be the first
neonatal Fc receptor (FcRn) blocker to treat CIDP
February 20, 2024, 7:00 AM CET
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 that the U.S. Food and
Drug Administration (FDA) has accepted for priority review a
supplemental Biologics License Application (sBLA) for VYVGART
Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for the
treatment of chronic inflammatory demyelinating polyneuropathy
(CIDP). The application has been granted a PDUFA target action date
of June 21, 2024.
“Today’s announcement brings us one step closer
to delivering the transformative innovation of VYVGART Hytrulo to
CIDP patients,” said Luc Truyen, Chief Medical Officer of argenx.
“CIDP is yet another example of an autoimmune disease that has not
been well understood, and for which there has been insufficient
innovation for patients. We chose to use a priority review voucher
to accelerate review of our submission because CIDP patients have
long been waiting for new treatment options. FDA’s acceptance of
the sBLA represents an important milestone in our continued drive
to bring novel treatments for rare, autoimmune diseases, and a
significant step forward for people whose lives have been
profoundly impacted by this devastating disease.”
The sBLA is supported by data from the ADHERE
study, the largest clinical trial of CIDP to date, evaluating the
safety and efficacy of subcutaneously administered VYVGART Hytrulo
in adults with CIDP. The study met its primary endpoint
(p=0.000039), demonstrating a 61% lower risk of relapse (HR: 0.39
95% CI: 0.25; 0.61) with VYVGART Hytrulo compared to placebo. In
the open-label Stage A of the study, 67% of patients showed
evidence of clinical improvement (ECI) following treatment with
VYVGART Hytrulo. Given the mechanism of action of VYVGART Hytrulo
as an FcRn blocker, the clinical results established that IgG
autoantibodies play a significant role in the underlying biology of
CIDP.
VYVGART Hytrulo was well-tolerated with a safety
profile that is consistent with prior clinical trials and the known
profile of VYVGART®. After completing ADHERE, 99% of eligible
patients (226/228) continued to the ADHERE-+ open-label extension
study.
About Chronic Inflammatory Demyelinating
PolyneuropathyChronic inflammatory demyelinating
polyneuropathy (CIDP) is a rare and serious autoimmune disease of
the peripheral nervous system. Although confirmation of disease
pathophysiology is still emerging, there is increasing evidence
that IgG antibodies play a key role in the damage to the peripheral
nerves. People with CIDP experience fatigue, muscle weakness and a
loss of feeling in their arms and legs that can get worse over time
or may come and go. These symptoms can significantly impair a
person's ability to function in their daily lives. Without
treatment, one-third of people living with CIDP will need a
wheelchair.
About VYVGART® Hytrulo
(efgartigimod alfa and hyaluronidase-qvfc)VYVGART
Hytrulo is a subcutaneous combination of efgartigimod alfa, a human
IgG1 antibody fragment marketed for intravenous use as VYVGART®,
and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s
ENHANZE® drug delivery technology to facilitate subcutaneous
injection delivery of biologics. In binding to the neonatal Fc
receptor (FcRn), VYVGART Hytrulo results in the reduction of
circulating IgG. It is the first-and-only approved FcRn blocker
administered by subcutaneous injection.
VYVGART Hytrulo is the proprietary name in the
U.S. for subcutaneous efgartigimod alfa and recombinant human
hyaluronidase PH20. It may be marketed under different proprietary
names following approval in other regions.
See FDA-approved Important Safety Information
below and full Prescribing Information for VYVGART
Hytrulo for additional information
Important Safety
Information
What is VYVGART® HYTRULO (efgartigimod
alfa and hyaluronidase-qvfc)?
VYVGART HYTRULO 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).
IMPORTANT SAFETY
INFORMATION
What is the most important information I
should know about VYVGART HYTRULO?
VYVGART HYTRULO may cause serious side effects,
including:
-
Infection. VYVGART HYTRULO may increase the
risk of infection. The most common infections for efgartigimod
alfa-fcab-treated patients were urinary tract and respiratory tract
infections. More patients on efgartigimod alfa-fcab vs placebo had
below normal levels for white blood cell counts, lymphocyte counts,
and neutrophil counts. The majority of infections and observed
lower white blood cell counts were mild to moderate in severity.
Your healthcare provider should check you for infections before
starting treatment, during treatment, and after treatment with
VYVGART HYTRULO. Tell your healthcare provider if you have any
history of infections. Tell your healthcare provider right away if
you have signs or symptoms of an infection during treatment with
VYVGART HYTRULO 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. If a serious
infection occurs, your doctor will treat your infection and may
even stop your VYVGART HYTRULO treatment until the infection has
resolved.
-
Undesirable immune reactions (hypersensitivity reactions). VYVGART HYTRULO and
efgartigimod alfa-fcab can cause the immune system to have
undesirable reactions such as rashes, swelling under the skin, and
shortness of breath. Hives were also observed in patients treated
with VYVGART HYTRULO. 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 HYTRULO
discontinuation. Your healthcare provider should monitor you during
and after treatment and discontinue VYVGART HYTRULO if needed. Tell
your healthcare provider immediately about any undesirable
reactions to VYVGART HYTRULO.
Before taking VYVGART HYTRULO, 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 healthcare
provider whether you need to receive age-appropriate immunizations
before initiation of a new treatment cycle with VYVGART HYTRULO.
The use of vaccines during VYVGART HYTRULO 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 HYTRULO.
- Are pregnant or plan to become
pregnant and are breastfeeding or plan to breastfeed.
Tell your healthcare 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 HYTRULO?
The most common side effects of efgartigimod
alfa-fcab-treated patients were respiratory tract infection,
headache, and urinary tract infection. Additional common side
effects of VYVGART HYTRULO are injection site reactions, including
rash, redness of the skin, itching sensation, bruising, pain, and
hives.
These are not all the possible side effects of
VYVGART HYTRULO. 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 HYTRULO and talk to your
doctor.
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 approved neonatal Fc
receptor (FcRn) blocker in the U.S., Japan, Israel, the EU, the UK,
Canada and China. 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.
Contacts
Media:
Ben PetokBpetok@argenx.com
Investors:
Alexandra Roy (US) aroy@argenx.com
Lynn Elton (EU) lelton@argenx.com
Forward-Looking Statements
The 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 “plans,” “aims,”
“believes,” “continues,” “hope,” “estimates,” “anticipates,”
“expects,” “intends,” “may,” “will,” “should,” or “commitment” and
include statements argenx makes concerning our advancement towards
the delivery of VYVGART Hytrulo to CIDP patients, our continued
drive to bring novel treatments for rare, autoimmune diseases, and
our goal of translating immunology breakthroughs into a world-class
portfolio of novel antibody-based medicines. 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, including but
not limited to, the results of our PDUFA review for VYVGART Hytrulo
to CIDP patients, expectations regarding the inherent uncertainties
associated with development of novel drug therapies, preclinical
and clinical trial and product development activities and
regulatory approval requirements, the acceptance of our products
and product candidates by our patients as safe, effective and
cost-effective, and the impact of governmental laws and regulations
on our business. 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 to 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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