Sanofi's Wayrilz Gets Orphan Drug Tag for IgG4-Related Disease in Japan

Sanofi SNY announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug designation to Waylirz (rilzabrutinib) for the treatment of IgG4-related disease (IgG4-RD). The drug is not yet approved for this indication in any market.

How Does the Orphan Tag Benefit Sanofi’s Drug Development?

Orphan drug designation is granted by the MHLW to therapies intended for the treatment of rare diseases or conditions affecting fewer than 50,000 people in Japan. The goal is to encourage drug development for rare diseases or conditions by offering incentives such as priority consultation and guidance, preferential tax treatment, subsidies and priority review.

For Sanofi, this designation provides Waylirz with important regulatory and financial advantages as it advances through clinical development for IgG4-RD, a rare disease affecting nearly any organ and may lead to progressive organ damage if left untreated. The orphan drug designation may help accelerate progress and reduce the development burden as Sanofi works to address this critical unmet need.

This marks the third orphan drug designation for rilzabrutinib in IgG4-RD, following similar designations previously granted in the United States and the European Union.

More on Sanofi’s Waylirz

A novel BTK inhibitor, Waylirz, is designed to treat several rare immune-mediated or inflammatory diseases by restoring immune balance via multi-immune modulation. The drug is currently approved for immune thrombocytopenia (ITP) in the United States and the European Union. A regulatory filing for a similar indication is under review in Japan.

The orphan drug designation granted by MHLW to Waylirz for the IgG4-RD indication was supported by positive data from a mid-stage study. Data from this study showed that 52-week treatment reduced disease flares and other disease markers and minimized the need for treatment with glucocorticoids.

Sanofi is currently evaluating Waylirz in late-stage studies for IgG4-RD, sickle cell disease (SCD) and warm autoimmune hemolytic anemia (wAIHA). The drug is being evaluated in mid-stage studies for asthma and chronic spontaneous urticaria (CSU).

Waylirz Faces Competition in Targeted Markets

The Sanofi drug faces competition across several of its targeted indications, although the competitive intensity varies by disease area and development stage.

In the already approved ITP indication, the drug competes with Amgen’s AMGN Nplate and Rigel Pharmaceuticals’ RIGL Tavalisse. Though both the AMGN and RIGL drugs are built on different mechanisms, they pose a significant threat to Wayrilz that is a more recent entrant in the market.

Concerning IgG4-RD, Waylirz, if approved, may face competition from already approved therapies like Amgen’s Uplizna.

With regard to CSU, the drug would compete with the blockbuster drug Dupixent, marketed jointly by Sanofi and Regeneron, which has a strong clinical profile and significant market share across several immunology indications.

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