AMGN's Uplizna Gets FDA Nod for 2nd Rare Autoimmune Disease Indication

Amgen AMGN announced that the FDA has approved its rare disease drug Uplizna (inebilizumab) for a new indication. The regulatory body has now approved Uplizna for the treatment of adults living with immunoglobulin G4-related disease (IgG4-RD), a rare immune-mediated inflammatory condition. This marks the second approved indication for the drug.

Uplizna is currently approved in the United States for treating adults with a rare autoimmune disease called neuromyelitis optica spectrum disorder.

Following the nod, Uplizna became the first and only drug approved by the FDA for the treatment of IgG4-RD, a chronic disease with a significant unmet medical need.

The FDA had previously granted a breakthrough therapy designation to Uplizna for the treatment of IgG4-RD.

Year to date, shares of Amgen have rallied 18.9% compared with the industry’s rise of 1.2%.

Uplizna's Nod for IgG4-RD Based on AMGN's MITIGATE Study

The latest FDA nod for the expanded use of Uplizna in IgG4-RD was based on data from the phase III MITIGATE study.

The study evaluated the safety and efficacy of Uplizna versus placebo in reducing the risk of flares in adults with IgG4-RD. The primary endpoint of the study was to check the time to first treat and adjudicate IgG4-RD flare.

Data from the MITIGATE study showed that treatment with Uplizna led to an 87% reduction in the risk of flares as compared to placebo.

As part of the study, 58.8% of participants who were treated with Uplizna achieved flare-free, corticosteroid-free, and complete remission versus 22.4% of participants who received a placebo at week 52.

Per management, the latest approval for Uplizna should help patients reduce the use of harmful steroids along with reducing the risk of flares.

AMGN Developing Uplizna for Generalized Myasthenia Gravis

Amgen has been evaluating Uplizna for treating generalized myasthenia gravis (gMG), another immune-mediated disease.

Last month, the company reported the latest data from the phase III MINT study that evaluated Uplizna in the gMG indication.

Data from the study showed that treatment with Uplizna “demonstrated durable and sustained efficacy” in AChR-positive gMG patients over 52 weeks.

Based on these results, Amgen intends to submit a regulatory filing for Uplizna with the FDA by the first half of 2025.

Uplizna was added to Amgen’s rare disease portfolio with the acquisition of Horizon Therapeutics in 2023. The drug generated sales worth $379 million in 2024. Successful label expansion into additional indications should drive Uplizna sales in future quarters.

AMGN's Zacks Rank & Stocks to Consider

Amgen 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 declined 1.9%.

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 0.6%.

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 13.8%.

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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