Halozyme Announces argenx's VYDURA with ENHANZE® was Granted Regulatory Approval in Japan for Chronic Inflammatory Demyelinating Polyneuropathy

Rhea-AI Summary

Halozyme Therapeutics (NASDAQ: HALO) announced that argenx's VYVDURA, co-formulated with Halozyme's ENHANZE® drug delivery technology, received regulatory approval in Japan for treating chronic inflammatory demyelinating polyneuropathy (CIDP) in adults. The treatment, administered as a weekly 30-90 second subcutaneous injection, is the first FcRn blocker approved for CIDP.

The approval is based on the ADHERE Study, which showed 69% of patients demonstrated clinical improvement, with a 61% reduction in relapse risk versus placebo. VYVDURA was previously approved in Japan for generalized myasthenia gravis (gMG) in January 2024 and received Orphan Drug designation for CIDP in March 2024.

Positive

• Regulatory approval in Japan expands market access for ENHANZE technology
• Clinical trial showed strong efficacy with 69% of patients showing improvement
• Significant 61% reduction in relapse risk versus placebo
• Second approved indication in Japan, following gMG approval in January 2024
• Orphan Drug designation received for CIDP indication

Negative

• None.

Insights

Medical Research Analyst

<p>The approval of VYVDURA in Japan represents a <b>significant therapeutic advancement</b> for CIDP patients. The ADHERE study data is particularly compelling, showing a <percent>61%</percent> reduction in relapse risk and clinical improvements in <percent>69%</percent> of patients. The self-administered subcutaneous format, enabled by ENHANZE®, dramatically improves the treatment experience compared to traditional lengthy infusions.</p><p>The dual indication strategy (CIDP and gMG) maximizes the commercial potential in Japan's rare disease market. The Orphan Drug designation provides additional market advantages and reflects the unmet medical need. The near-universal enrollment in the extension study (ADHERE+) strongly validates the treatment's value proposition to patients.</p>

Financial Analyst

<p>This approval strengthens Halozyme's revenue potential through royalties and milestone payments from argenx. With VYVDURA now approved for both CIDP and gMG in Japan, the <b>addressable market expands significantly</b>. Japan represents the world's third-largest pharmaceutical market and rare disease treatments typically command premium pricing.</p><p>The ENHANZE® technology platform continues to demonstrate its strategic value, enabling improved drug delivery methods that benefit both patients and healthcare systems. This success reinforces Halozyme's business model and validates their technology's applicability across multiple therapeutic areas, potentially attracting more licensing partnerships.</p>

SAN DIEGO, Dec. 30, 2024 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) (Halozyme) today announced that argenx's VYVDURA (efgartigimod alfa and hyaluronidase-qvfc), which is co-formulated with Halozyme's ENHANZE^® drug delivery technology, was granted regulatory approval by Japan's Ministry of Health, Labour and Welfare (MHLW) for adults with chronic inflammatory demyelinating polyneuropathy (CIDP). VYVDURA was approved for CIDP as a once weekly 30-to-90 second subcutaneous injection, which can be self-administered at home, and is the first and only neonatal Fc receptor (FcRn) blocker approved for the treatment of CIDP.

"We are pleased that VYVDURA, with our innovative ENHANZE drug delivery technology, is now approved for two indications in Japan, enabling greater flexibility and optionality for generalized myasthenia gravis and CIDP patients," said Dr. Helen Torley, president and chief executive officer of Halozyme. "We look forward to VYVDURA being a new treatment option for an even broader number of patients in Japan."

The MHLW approval is based on the ADHERE Study, the largest clinical trial to date studying CIDP. In the ADHERE study, 69% (221/322) of patients treated with VYVDURA, regardless of prior treatment, demonstrated evidence of clinical improvement, including improvements in mobility, function and strength. ADHERE met its primary endpoint (p<0.0001) demonstrating a 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in the risk of relapse versus placebo. Ninety-nine percent of trial participants elected to participate in the ADHERE+ open-label extension. The safety results were generally consistent with the known safety profile of VYVDURA in previous clinical studies and real-world use.

VYVDURA was also approved by the MHLW for manufacturing and marketing in January 2024 and launched in April 2024 for the treatment of generalized myasthenia gravis (gMG). In March 2024, VYVDURA was designated as an Orphan Drug for the treatment of CIDP by the MHLW.

About Halozyme

Halozyme is a biopharmaceutical company advancing disruptive solutions to improve patient experiences and outcomes for emerging and established therapies. As the innovators of ENHANZE^® drug delivery technology with the proprietary enzyme rHuPH20, Halozyme's commercially-validated solution is used to facilitate the subcutaneous delivery of injected drugs and fluids, with the goal of improving the patient experience with rapid subcutaneous delivery and reduced treatment burden. Having touched more than 800,000 patient lives in post-marketing use in nine commercialized products across more than 100 global markets, Halozyme has licensed its ENHANZE^® technology to leading pharmaceutical and biotechnology companies including Roche, Takeda, Pfizer, Janssen, AbbVie, Eli Lilly, Bristol-Myers Squibb, argenx, ViiV Healthcare, Chugai Pharmaceutical and Acumen Pharmaceuticals.

Halozyme also develops, manufactures and commercializes, for itself or with partners, drug-device combination products using its advanced auto-injector technologies that are designed to provide commercial or functional advantages such as improved convenience, reliability and tolerability, and enhanced patient comfort and adherence. The Company has two commercial proprietary products, Hylenex^® and XYOSTED^®, partnered commercial products and ongoing product development programs with Teva Pharmaceuticals and Idorsia Pharmaceuticals.

Halozyme is headquartered in San Diego, CA and has offices in Ewing, NJ and Minnetonka, MN. Minnetonka is also the site of its operations facility.

For more information visit www.halozyme.com and connect with us on LinkedIn and Twitter.

Safe Harbor Statement

In addition to historical information, the statements set forth above include forward-looking statements including, without limitation, statements concerning the possible activity, benefits and attributes of ENHANZE^®, the possible method of action of ENHANZE^®, its potential application to aid in the dispersion and absorption of other injected therapeutic drugs, and statements concerning certain other potential benefits of ENHANZE^® including facilitating more rapid delivery of injectable medications through subcutaneous delivery and potentially lowering the treatment burden for patients, including offering flexibility to receive treatment in more convenient locations and broadening the treatment options for the indications referred to in this press release. These forward-looking statements involve risks and uncertainties that could cause actual results to differ materially from those in the forward-looking statements. The forward-looking statements are typically, but not always, identified through use of the words "expect," "believe," "enable," "may," "will," "could," "intends," "estimate," "anticipate," "plan," "predict," "probable," "potential," "possible," "should," "continue," and other words of similar meaning. Actual results could differ materially from the expectations contained in forward-looking statements as a result of several factors, including unexpected results or delays in launch or commercialization of our partner's product for the indication referred to in this press release, unexpected adverse events or patient experiences or outcomes from being treated with the ENHANZE^® co-formulated treatment referred to in this press release, and competitive conditions. These and other factors that may result in differences are discussed in greater detail in Halozyme's most recent Annual and Quarterly Reports filed with the Securities and Exchange Commission. Except as required by law, Halozyme undertakes no duty to update forward-looking statements to reflect events after the date of this release.

Contacts:

Tram Bui
VP, Investor Relations and Corporate Communications
609-359-3016
tbui@halozyme.com

Samantha Gaspar
Teneo
212-886-9356
samantha.gaspar@teneo.com

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SOURCE Halozyme Therapeutics, Inc.

FAQ

What was the efficacy rate of VYVDURA (HALO) in the ADHERE Study for CIDP treatment?

In the ADHERE Study, 69% (221/322) of patients treated with VYVDURA showed evidence of clinical improvement, including improvements in mobility, function and strength.

When did VYVDURA with HALO's ENHANZE technology receive approval for CIDP in Japan?

VYVDURA received regulatory approval from Japan's Ministry of Health, Labour and Welfare (MHLW) for CIDP treatment in December 2024.

How is VYVDURA with HALO's ENHANZE technology administered for CIDP treatment?

VYVDURA is administered as a once weekly 30-to-90 second subcutaneous injection, which can be self-administered at home.

What indications has VYVDURA (HALO) been approved for in Japan?

VYVDURA has been approved in Japan for two indications: chronic inflammatory demyelinating polyneuropathy (CIDP) and generalized myasthenia gravis (gMG).

What was the risk reduction in relapse for VYVDURA (HALO) compared to placebo in CIDP treatment?

VYVDURA demonstrated a 61% reduction in the risk of relapse versus placebo in the ADHERE study.