FDA approves Vyvgart Hytrulo under the skin as a treatment for gMG |…

The US Food and Drug Administration (FDA) has approved the subcutaneous formulation of efgartigimod Argenxs, now called Vyvgart Hytrulo, for the treatment of adults with generalized myasthenia gravis (GMG).

The newly approved therapy is indicated for gMG patients positive for antibodies to the acetylcholine receptor (AChR), the most common type of autoreactive antibodies that drive MG.

Vyvgart Hytrulo is a subcutaneous or subcutaneous combination product of efgartigimod alfa, the active agent of Argenxs Vyvgart intravenous gMG therapy, and a recombinant or man-made human hyaluronidase enzyme PH20. This enzyme plays a key role in how the treatment is administered subcutaneously.

With this approval, Vyvgart Hytrulo has now become the first FDA-approved gMG therapy to be delivered via a subcutaneous injection, which takes less than two minutes up to 90 seconds to administer. By comparison, intravenous or intravenous therapy takes about an hour.

[The FDAs] The approval of Vyvgart Hytrulo is another significant milestone in our journey to redefine what well-controlled means for gMG patients, Luc Truyen, MD, PhD, medical director of Argenxs, said in a company news release, adding, With our extensive gMG offering of both a first-class brew and [subcutaneous] injection, we continue to offer an individualized treatment approach and [the] ability to remain symptom-free while providing patients with options for how and where they want to seek treatment.

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Approval acclaimed by patients, lawyers

As stated in its label, Vyvgart Hytrulo should be administered by a healthcare professional. The drug is to be administered as a single subcutaneous injection containing a fixed dose of 1,008 mg of efgartigimod alfa over 30 to 90 seconds, in cycles of once-weekly injections lasting four weeks.

In addition to the much shorter administration time, Vyvgart Hytrulo also gives patients the opportunity to receive the drug at home, an option not generally possible with the intravenous formulation.

Such advances are especially noteworthy, according to Sharon Hesterlee, PhD, chief research officer of the Muscular Dystrophy Association (MDA), given that few treatment options were available a decade ago.

The approval of an additional formulation of Vyvgart is another important step toward treating gMG, a disease that, until a few years ago, had no approved therapies, Hesterlee said in an MDA news release.

“Now those living with gMG will have the opportunity to receive a subcutaneous injection of an effective disease-modifying therapy,” said Hesterlee.

The decision has been well received by the patient community, as well as several patient organizations and associations.

The availability of another treatment option for Argenx gMG, now with subcutaneous administration, represents a significant advance for the patient community, said Allison Foss, executive director of the Myasthenia Gravis Association.

“Patients now have the opportunity to receive care in an infusion center, at home or in a doctor’s office, offering greater flexibility and freedom of choice that can make the daily lives of MG patients and their caregivers easier” Foss said.

Stephanie Madole is a mother of two daughters with gMG. While both are on other therapies, she called the FDA approval “an exciting step forward.”

I feel Vyvgart’s new formulation has the potential to be more convenient, which will definitely make a difference for many families, Madole said, adding, “Continued research and more treatment options are imperative.

The new formulation replaces a one-hour infusion with an injection given at home; which is important for families like ours who drive 8 hours round trip for infusions and our girls miss school and the days where they can only spend time as children,” Madole said.

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Vyvgart Hytrulo likely available in July

According to Argenx, Vyvgart Hytrulo is expected to be available to patients in the United States in July. As part of its commitment to support patient access to its medicines, Argenx has decided to price Vyvgart Hytrulo on par with its previously approved gMG therapy, Vyvgart.

In addition, the company is making Vyvgart’s personalized patient support program, My Vyvgart Path, available to patients starting treatment with Vyvgart Hytrulo. The program includes helpful resources and information about disease education, as well as financial assistance programs.

The FDA’s decision comes about nine months after Argenx initially filed for regulatory filing and about seven months after the agency granted priority review. Earlier this year, the FDA delayed the review deadline, initially set for mid-March, by three months so that it could thoroughly analyze the additional information submitted by Argenx.

Subcutaneous efgartigimod is also under regulatory review in both the European Union and Japan. A decision in the EU is expected later this year, with action in the Asian nation anticipated early next year.

In MG, the body’s immune system produces self-reactive antibodies that mistakenly attack healthy proteins at the neuromuscular junctions where nerve and muscle cells communicate.

Efgartigimod is an antibody fragment that binds to and blocks the activity of the neonatal Fc receptor, a protein that normally helps prevent the destruction of antibodies circulating in the bloodstream. Thus, it is expected to increase the rate at which the autoreactive antibodies that drive MG are broken down, lowering their blood levels and ultimately relieving the symptoms of the disease.

Vyvgart is an approved formulation of efgartigimod that is delivered directly into the bloodstream in hour-long infusions. A treatment cycle consists of weekly infusions given over four weeks, with further treatment courses given based on the patient’s clinical response.

The therapy is approved in the United States and other regions for adults with gMG-positive AChR antibodies. Such patients make up approximately 85% of the total gMG population.

Vyvgart Hytrulo was designed to provide an easier-to-administer formulation of efgartigimod than Vyvgart. It was developed using a technology from Halozyme Therapeutics called ENHANZE. Such technology facilitates the delivery of biological therapies that are typically administered by infusion directly into the bloodstream. It is based on the use of a proprietary enzyme that breaks down an important component of skin tissue, facilitating the dispersion and absorption of medicines administered by subcutaneous injection.

We are pleased that Argenx has received FDA approval for the subcutaneous form of efgartigimod, which reinforces their commitment to the patient community with expanded treatment options that offer flexibility to patients. Helen TorleyHalozymes chairman and chief executive officer, said in another news release.

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New formulation of efgartigimod comparable to Vyvgart in studies

The approval of the therapy was supported primarily by data from the international Phase 3 clinical study ADAPT-SC (NCT04735432). That trial tested Vyvgart Hytrulo against Vyvgart in 110 adults with gMG.

Participants, most of whom were AChR antibody positive, were randomly assigned to receive weekly treatment with Vyvgart Hytrulo (1,000 mg) or Vyvgart (10 mg/kg) for four weeks or approximately one cycle. of treatment.

The results showed that Vyvgart Hytrulow was, at least, not inferior to Vyvgart in its ability to lower levels of immunoglobulin G (IgG), a class of antibodies that includes those implicated in MG after 29 days of treatment (an average drop of 66 .4% vs. 62.2%). Comparable results were observed between the two therapies regardless of patients’ anti-AChR antibody status.

More than two-thirds (69.1%) of Vyvgart Hytrulo-treated patients experienced a clinically meaningful reduction in the impact of the disease on daily living, as evidenced by a drop of two or more points in the Activities of Daily Living MG score for at least four consecutive weeks.

About as many patients treated with Vyvgart Hytrulo (65.5%) showed equally sustained reduction in disease severity, as evidenced by at least a three-point drop in quantitative MG score.

More than a third of patients (37%) were symptom-free after a course of treatment with Vyvgart Hytrulo.

The therapy was generally well tolerated, with a safety profile comparable to that of Vyvgart. The most frequently reported side effects with the new formulation were mild to moderate injection site reactions, all of which resolved over time.

Now, with the approval of Vyvgart Hytrulo, we have a broad offering of gMG treatments with both [intravenous] AND [subcutaneous] administration options and can select based on patient needs and preferences without sacrificing clinical benefit or safety.

Vyvgart clinical trials continue to show significant benefits for patients with a favorable safety profile and clear improvements in gMG disease scores. Now, with the approval of Vyvgart Hytrulo, we have a broad offering of gMG treatments with both [intravenous] AND [subcutaneous] administration options and can select based on patient needs and preferences without sacrificing clinical benefit or safety, said James F. Howard Jr., MD, professor of neurology at the University of North Carolina at Chapel Hill School of Medicine and Principal Investigator in ADAPT-SC.

The majority of patients who completed ADAPT-SC chose to participate in an open-label extension study, called ADAPT-SC+ (NCT04818671), in which all will receive Vyvgart Hytrulo for up to two years. If necessary, repeated courses of treatment will be given, separated by about a month or more.

Interim six-monthly data showed that each Vyvgart Hytrulocycle was associated with a reduction in disease severity and that no new safety issues were identified with longer treatment.

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