Mirum (MIRM) Gets EC Approval for Livmarli Label Expansion

Mirum Pharmaceuticals, Inc. MIRM announced that the European Commission (“EC”) has approved its lead drug, Livmarli (maralixibat) oral solution for a new indication. Shares of the company were up 4.3% on Jul 8 following the announcement of the news.

The EC has now approved Livmarli for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged three months and above.

The latest approval was based on data from the phase III MARCH study, which evaluated Livmarli in patients across a range of genetic PFIC types and unidentified mutational status.

Data from the study showed that treatment with Livmarli led to a statistically significant reduction in pruritus and serum bile acids compared with placebo. Treatment with Livmarli also significantly improved total bilirubin and growth versus placebo.

The Committee for Medicinal Products for Human Use rendered a positive opinion for Livmarli for the treatment of PFIC in patients aged three months and older in May.

The FDA approved Livmarli oral solution for the treatment of cholestatic pruritus in patients aged five years and older with PFIC in March. PFIC is a rare genetic disorder that causes liver failure.

Shares of Mirum have rallied 21.6% so far this year against the industry’s decline of 7.1%.

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Livmarli is an orally administered ileal bile acid transporter inhibitor. Livmarli is already approved in the United States and EU as the only medication for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged three months and older and two months and older, respectively.

MIRM has also submitted an additional supplemental new drug application to the FDA seeking label expansion for a higher concentration formulation of Livmarli, which was used in the MARCH study to include treatment of younger patients with PFIC. Feedback from the regulatory body is expected later in 2024.

In the first quarter of 2024, Livmarli generated sales worth $42.8 million, reflecting an increase of almost 47% on a year-over-year basis. The approval for the PFIC indication should drive sales further in 2024.

Zacks Rank & Stocks to Consider

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

Some better-ranked stocks in the healthcare sector are Acrivon Therapeutics, Inc. ACRV, Anavex Life Sciences Corp. AVXL and RAPT Therapeutics, Inc. RAPT, 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 Acrivon Therapeutics’ 2024 loss per share have narrowed from $3.05 to $2.47. Loss per share estimates for 2025 have narrowed from $3.04 to $2.55. Year to date, ACRV shares have rallied 43.9%.

ACRV’s earnings beat estimates in three of the trailing four quarters and missed the same on the remaining one occasion, the average surprise being 3.56%.

In the past 60 days, estimates for Anavex Life Sciences’ 2024 loss per share have narrowed from 57 cents to 55 cents, while loss per share estimates for 2025 have narrowed from 87 cents to 80 cents. Year to date, AVXL shares have declined 51.6%.

Anavex Life Sciences’ earnings beat estimates in three of the trailing four quarters and met the same once, the average surprise being 17.33%.

In the past 60 days, estimates for RAPT Therapeutics’ 2024 loss per share have narrowed from $3.19 to $2.93. Loss per share estimates for 2025 have narrowed from $2.40 to $2.05. Year to date, RAPT shares have declined 88.2%.

RAPT’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average surprise being 3.19%.

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