SNY's Rilzabrutinib Gets FDA's Orphan Drug Tag for Two Rare Diseases

Sanofi SNY announced that the FDA has granted orphan drug designation to its investigational BTK inhibitor, rilzabrutinib, for the treatment of warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD).

The candidate is being developed in separate mid-stage studies for treating wAIHA and IgG4-RD, two rare diseases.

Currently, there are no approved therapies for treating wAIHA and IgG4-RD. Both diseases have a significant unmet medical need.

The FDA grants orphan drug designation to support the development of medicines for rare disorders that affect less than 200,000 patients in the United States.

SNY's Price Performance

Year to date, shares of Sanofi have gained 11.9% compared with the industry’s rise of 0.8%.

Data from a phase IIb study evaluating rilzabrutinib for wAIHA showed that treatment with the candidate demonstrated clinically meaningful outcomes on response rate and disease markers.

Per management, data from a phase IIa study evaluating rilzabrutinib in IgG4-RD patients showed that treatment with the candidate for 52 weeks led to a reduction in disease flare, other disease markers and glucocorticoid sparing. Further details from the same will be announced at an upcoming medical conference.

SNY's Rilzabrutinib Under Review for Immune Thrombocytopenia

Sanofi has developed rilzabrutinib for treating immune thrombocytopenia (“ITP”), another rare, complex autoimmune disorder.

A regulatory application seeking approval for rilzabrutinib for treating ITP is currently under review in the United States. A final decision from the FDA is expected on Aug. 29, 2025. The regulatory body has previously granted a fast-track designation and an orphan drug designation to rilzabrutinib for treating ITP in the United States.

Besides the United States, rilzabrutinib is also under review in the European Union and China as a potential treatment for ITP.

Rilzabrutinib was added to Sanofi’s pipeline with the acquisition of Principia Biopharma. The candidate is also being developed in mid-stage studies for other immune-mediated diseases like asthma and chronic spontaneous urticaria.

SNY's Zacks Rank & Stocks to Consider

Sanofi currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Jazz Pharmaceuticals, Inc. JAZZ, Dynavax Technologies Corporation DVAX and Krystal Biotech, Inc. KRYS, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

In the past 60 days, estimates for Jazz Pharmaceuticals’ earnings per share have increased from $22.06 to $23.33 for 2025. During the same time, earnings per share estimates for 2026 have increased from $23.13 to $23.35. Year to date, shares of JAZZ have risen 2.7%.

JAZZ’s earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 3.20%.

In the past 60 days, estimates for Dynavax’s earnings per share have increased from 32 cents to 33 cents for 2025. During the same time, earnings per share estimates have increased from 49 cents to 57 cents for 2026. Year to date, shares of DVAX have increased 0.8%.

DVAX’s earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 9.58%.

In the past 60 days, estimates for Krystal Biotech’s earnings per share have increased from $5.40 to $7.06 for 2025. During the same time, earnings per share estimates for 2026 have increased from $9.15 to $11. Year to date, shares of KRYS have rallied 15.6%.

KRYS’ earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 3.29%.

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