As efgartigimod gains traction in myasthenia gravis, argenx is out to prove its antibody fragment is no one hit wonder. Now, armed with a phase 3 win in primary immune thrombocytopenia (ITP), the company is bolstering its thesis that the neonatal-receptor-binding drug can drive down antibodies implicated across a host of autoimmune diseases.

ASH: With immune thrombocytopenia win, argenx proves efgartigimod's no one-trick pony in autoimmune disease

The FDA granted argenx Priority Review status for the company’s Biologics License Application for SC efgartigimod for generalized myasthenia gravis (gMG).

The drug is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s Enhanze drug delivery system. Enhanze helps deliver biologics subcutaneously that are usually administered via intravenous infusion.

Argenx has filed for FDA approval of subcutaneous efgartigimod, keeping it on track to start selling the sibling of its existing intravenous generalized myasthenia gravis (gMG) treatment Vyvgart.

A pharmaceutical giant reported a Phase 3 success for a potential blockbuster drug, while four other companies reported disappointing data from their respective clinical trials. Our recap of data readouts and regulatory news also includes one FDA rejection.

Last year Argenx bagged approval for the first FcRn antagonist, efgartigimod, trademarked as the intravenously delivered Vyvgart. Now a subcutaneous version is heading to regulators by year end, following success in the pivotal Adapt-SC trial in generalised myasthenia gravis.

There are some lingering questions about the incidence of adverse events with the subcutaneous formulation. But assuming that these will not worry the FDA, the big unknown at present is how Argenx will choose to price this more convenient option – something that, for now, it will not discuss.

The most valuable US approval decision left for 2021 concerns Argenx’s efgartigimod in myasthenia gravis. A green light is expected, but questions remain over how broad the label might be.

Calliditas could also gain its first US approval after a three-month delay with Nefecon. Meanwhile, Merck & Co’s Keytruda could bag its eighth FDA nod this year alone, this time in adjuvant melanoma.

Looking to close out 2021 with FDA approvals stand four potential blockbusters from the likes of Argenx, UCB, Pfizer and Roche, according to Evaluate Pharma. Those meds combined are worth roughly $7.1 billion in sales cumulatively by 2026, according to Evaluate’s estimates. 

The US FDA’s verdict on Biogen’s aducanumab is one of the biggest events on biopharma’s 2021 calendar. And the Alzheimer’s antibody owns another accolade: of the novel projects slated to reach the market this year, aducanumab attracts the sellside's highest revenue forecasts.

Big Pharma friend argenx has snapped up an FDA priority review voucher from Bayer for $98 million.

The little biotech, which has a series of deals across Big Pharma, will use the voucher, which can speed up the regulatory process for a new drug and get it approved more quickly, for its late-stage drug efgartigimod—but not in the indication you might think.

A phase 3 trial of Argenx’s efgartigimod in generalized myasthenia gravis (gMG) patients has hit its primary endpoint, setting the European biotech up to file for FDA approval by the end of the year.