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argenx Receives Notification of PDUFA Date Extension for SC Efgartigimod

Key Takeaway: argenx has received notification from the U.S. FDA regarding an extension of the review period for its Biologics License Application for subcutaneous efgartigimod, now set for June 20, 2023. This extension was prompted by a major amendment related to the ongoing review process. Importantly, the FDA has not requested any additional data or studies at this time. The company remains optimistic about efgartigimod's profile and its potential to treat generalized myasthenia gravis.

Market Sentiment Analysis

POSITIVE FACTORS

  • FDA has extended the review period for SC efgartigimod, indicating thorough consideration.
  • No additional data or studies have been requested, suggesting confidence in submitted materials.
  • argenx is optimistic about the product's profile and the strength of the dataset supporting it.

Full Press Release Details

argenx Receives Notification of PDUFA Date Extension
Netherlands - argenx (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 extended the
review of the Biologics License Application (BLA) for subcutaneous (SC) efgartigimod (1000mg efgartigimod-PH20) for the treatment of adult
patients with generalized myasthenia gravis (gMG) to June 20, 2023.
The FDA notified argenx on January 26, 2023
that information submitted in connection with the ongoing review of the SC efgartigimod BLA constituted a major amendment and requires
an extension to allow sufficient time to review. No additional data or studies have been requested at this time.
"We are confident in the profile of SC
efgartigimod and the strength of the ADAPT-SC dataset showing noninferiority of our subcutaneous product to VYVGART . We will
continue to work closely with the FDA as it completes its review to bring this important medicine to people living with gMG,"
said Luc Truyen, M.D., Ph.D., Chief Medical Officer, argenx.
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 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 months, where muscles throughout the
body may be affected. Patients with confirmed AChR antibodies account for approximately 85% of the total gMG population.
argenx 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., the EU and Japan.
For further information, please contact:
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 "believes," "hope," "estimates," "anticipates," "expects,"
"intends," "may," "will," or "should" and include statements argenx makes concerning the
profile of subcutaneous (SC) efgartigimod and the strength of the ADAPT-SC dataset; the initiation, timing, progress and results of its
anticipated clinical development and regulatory milestones and plans; and the timing and outcome of regulatory filings and regulatory
approvals. 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.

Frequently Asked Questions

What is the PDUFA date extension for argenx?

The PDUFA date for argenx's SC efgartigimod review has been extended to June 20, 2023.

Why was the BLA review for efgartigimod extended?

The FDA deemed the submitted information a major amendment, requiring more review time.

What condition does efgartigimod aim to treat?

Efgartigimod is aimed at treating adult patients with generalized myasthenia gravis.

How does efgartigimod work?

Efgartigimod reduces pathogenic IgG antibodies by blocking their recycling process.

What is generalized myasthenia gravis (gMG)?

gMG is a chronic autoimmune disease causing muscle weakness due to IgG autoantibodies.

Last updated: Jan 27, 2023