FOSTER CITY, Calif. - Mirum Pharmaceuticals, Inc. (NASDAQ: NASDAQ:MIRM) received a positive recommendation from the Canadian Agency for Drugs and Technologies in Health (CADTH) for the public reimbursement of LIVMARLI® (maralixibat oral solution).
This decision follows LIVMARLI's prior approval by Health Canada in 2023 for treating cholestatic pruritus in patients with Alagille Syndrome (ALGS), a rare genetic liver disorder.
The recommendation is a significant development for patients with ALGS, who experience severe itching due to cholestasis, a condition where bile acids build up in the liver. Chris Peetz, CEO of Mirum, emphasized the importance of making the treatment widely available, noting the severe impact of ALGS, which typically begins in childhood.
The CADTH's endorsement is based on data from the ICONIC study, which demonstrated significant reductions in pruritus and serum bile acids in patients, with effects sustained over several years. LIVMARLI is the sole approved medication for cholestatic pruritus in ALGS patients in Canada and is also authorized for use in the United States and Europe.
ALGS affects the bile ducts and can lead to progressive liver disease. The disorder is often indicated for liver transplants due to pruritus. LIVMARLI, an ileal bile acid transporter (IBAT) inhibitor, is administered orally once a day. It is approved for ALGS patients three months and older in the U.S., two months and older in Europe, and by Health Canada.
Mirum Pharmaceuticals is focused on developing treatments for rare diseases. In addition to LIVMARLI, the company has two other approved medications and investigational treatments in its pipeline for other liver conditions.
The company cautions that this press release contains forward-looking statements about the potential commercial success of LIVMARLI and its effectiveness beyond treating pruritus. These statements are subject to risks and uncertainties, and actual results may differ materially.
This article is based on a press release statement.
InvestingPro Insights
As Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) continues to make strides in providing treatments for rare diseases, recent data and insights from InvestingPro paint a detailed financial picture of the company. Despite the clinical success of LIVMARLI, analysts are cautious about the company's profitability in the near term.
According to InvestingPro Tips, analysts have revised their earnings estimates downwards and do not expect Mirum to be profitable this year. This aligns with the company's current financial metrics, which indicate that Mirum has not been profitable over the last twelve months as of Q4 2023.
On a positive note, Mirum's liquid assets exceed its short-term obligations, suggesting a degree of financial stability in managing its immediate liabilities. Moreover, the company operates with a moderate level of debt, which may provide some flexibility in its operations and investment strategies. It is important to note that Mirum does not pay a dividend to shareholders, which is common for companies focused on growth and reinvestment.
Taking a closer look at the InvestingPro Data, Mirum's market capitalization stands at approximately $1.2 billion, reflecting the market's valuation of the company. The revenue growth has been impressive, with a surge of 141.85% over the last twelve months as of Q4 2023, which could be indicative of the company's potential and the market's response to its treatments like LIVMARLI.
Yet, the P/E ratio is currently negative at -7.32, underscoring the lack of profitability in the recent period. Investors should also be aware that the company's Price / Book ratio is 4.81, which might suggest a premium compared to its book value.
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