Mirum Pharmaceuticals, Inc. (NASDAQ: NASDAQ:MIRM) announced Thursday that the U.S. Food and Drug Administration (FDA) has approved an expanded indication for its drug LIVMARLI® (maralixibat) oral solution.
The approved label expansion allows for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) aged 12 months and older.
The decision by the FDA follows the results of the MARCH Phase 3 study, which evaluated a higher concentration formulation of LIVMARLI.
"The launch of LIVMARLI in PFIC is going well and we are thrilled that it will now be available for patients 12 months and older," said Chris Peetz, chief executive officer at Mirum, adding, "PFIC is generally diagnosed when children are young, and initiating treatment quickly after diagnosis will help to ensure they have fewer days suffering from pruritus associated with this rare liver disease."
LIVMARLI is an ileal bile acid transporter (IBAT) inhibitor that has been previously approved for pediatric cholestatic liver diseases, including Alagille syndrome (ALGS). In the United States, it is authorized for use in ALGS patients three months of age and older, while in Europe, the drug is approved for patients two months and older.
The medication has also received Breakthrough Therapy designation for both ALGS and PFIC type 2, as well as an orphan designation for these conditions.
However, LIVMARLI is not recommended for PFIC type 2 patients with a severe defect in the bile salt export pump (BSEP) protein. Side effects of the drug can include liver injury and gastrointestinal issues such as diarrhea and stomach pain.
Additionally, patients may experience Fat Soluble Vitamin (FSV) Deficiency, which can be exacerbated by the treatment.
Mirum Pharmaceuticals specializes in developing treatments for rare diseases affecting both children and adults. Besides LIVMARLI, the company's portfolio includes CHOLBAM® (cholic acid) capsules and CHENODAL® (chenodiol) tablets. Mirum is also advancing investigational treatments for other liver diseases, such as primary sclerosing cholangitis and primary biliary cholangitis, through its clinical trials.
Meanwhile, Mirum Pharmaceuticals has seen a series of positive developments. The company reported robust financial performance with $69 million in total net product revenue in the first quarter of 2024, of which Livmarli contributed $43 million. The company's revenue forecast for 2024 is between $310 million and $320 million. Analyst firms such as JMP Securities, Stifel, and H.C. Wainwright have expressed confidence in the company's performance, with JMP Securities projecting a revenue estimate of $316 million.
Mirum Pharmaceuticals also received European Union approval for its drug, Livmarli, for progressive familial intrahepatic cholestasis (PFIC) patients aged three months and older. This approval is expected to aid in broadening the drug's current U.S. label. Additionally, the company has submitted a supplemental New Drug Application for a higher concentration formulation of Livmarli, aiming to extend its use to younger patients.
InvestingPro Insights
Mirum Pharmaceuticals (NASDAQ: MIRM), following the FDA's nod for an expanded indication of its drug LIVMARLI®, shows promise as it continues to cater to rare liver diseases. The company's market capitalization stands at a robust $1.84 billion, reflecting investor confidence in its growth trajectory. Notably, Mirum's revenue has seen a significant uptick, with a remarkable 133.9% growth over the last twelve months as of Q1 2024, and an even more impressive quarterly revenue growth of 119.07% in Q1 2024. This financial growth could be indicative of the company's potential to capitalize on its recent FDA approval and expand its market reach.
While Mirum Pharmaceuticals operates with a moderate level of debt, its liquid assets surpass short-term obligations, suggesting a solid financial position to manage its liabilities and invest in future growth. However, investors should be aware that analysts have revised their earnings downwards for the upcoming period and do not anticipate the company to be profitable this year, as indicated by the negative P/E ratio of -11.61. Additionally, Mirum's stock has been performing well, trading near its 52-week high and exhibiting a strong return over the last year with a 55.97% price total return.
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