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FOSTER CITY, Calif. - Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a $2.53 billion market cap biopharmaceutical company that has seen its stock surge nearly 89% over the past year, has announced the U.S. Food and Drug Administration’s approval of CTEXLI™ (chenodiol) tablets for the treatment of cerebrotendinous xanthomatosis (CTX) in adults, making it the first and only approved medication for this rare and progressive disease. According to InvestingPro data, the company has demonstrated remarkable revenue growth of 112% in the last twelve months.
The FDA’s decision was based on the Phase 3 RESTORE study, which showed a highly significant reduction in urine bile alcohols, a primary disease marker, in patients treated with CTEXLI compared to those receiving a placebo. The study results indicated a 20-fold difference in urine 23S-pentol levels between the two groups. With the stock trading near its 52-week high of $54.23, analysts maintain a strong buy consensus with price targets ranging from $50 to $74.
CTX is characterized by the buildup of cholestanol, leading to severe neurological dysfunction and other symptoms due to a deficiency in the bile acid chenodeoxycholic acid (CDCA). The RESTORE study demonstrated that CTEXLI treatment improved not only urine bile alcohol levels but also serum cholestanol levels, suggesting a reduction in disease progression and symptoms.
Chris Peetz, CEO of Mirum, expressed hope that the approval would lead to earlier diagnosis and treatment, potentially sparing patients from debilitating symptoms. Dr. Ernst J. Schaefer of Tufts University School of Medicine and Jean Pickford, executive director of the CTX Alliance, echoed the sentiment, highlighting the importance of early intervention in CTX.
CTEXLI, which is another name for CDCA, was originally approved for gallbladder stone treatment and has now been repurposed for CTX. Patients can access the medication through Mirum Access Plus, the company’s patient support program.
While CTEXLI has been approved based on its efficacy, it also comes with potential side effects, including liver injury and common adverse reactions such as diarrhea and headache. Patients are advised to undergo liver function testing before and during treatment.
This approval marks a significant milestone for Mirum Pharmaceuticals, which specializes in rare diseases and has other approved medications such as LIVMARLI® and CHOLBAM®. The company continues to develop treatments for a range of rare conditions, with a focus on delivering transformative therapies.
The information for this article is based on a press release statement from Mirum Pharmaceuticals.
In other recent news, Mirum Pharmaceuticals announced preliminary and unaudited 2024 financial results, reporting net product sales of approximately $336 million, surpassing its sales guidance. The company projects 2025 global net product sales to be between $420 million and $435 million. The financial strength of Mirum is underscored by a year-end cash balance of roughly $287 million and positive cash flow from operations in the third quarter of 2024. Additionally, Mirum Pharmaceuticals has expanded its commercial access to 30 countries, with successful product launches in major European markets.
In terms of regulatory advancements, the FDA has approved LIVMARLI for cholestatic pruritus in progressive familial intrahepatic cholestasis (PFIC) patients aged 12 months and older. The company also anticipates the completion of the VISTAS study of volixibat in the second half of 2025. Mirum’s New Drug Application (NDA) for chenodiol is under FDA review, with a Prescription Drug User Fee Act (PDUFA) date set for March 28, 2025, for the treatment of cerebrotendinous xanthomatosis (CTX).
Meanwhile, JMP Securities reaffirmed its Market Outperform rating and $74 price target for Mirum Pharmaceuticals, emphasizing confidence in Ctexli, a therapy for CTX. This confidence is bolstered by strong Phase 3 RESTORE data and the drug’s FDA medical necessity status, which significantly contributes to the company’s revenues. The firm’s analyst, Jonathan Wolleben, noted that increasing the probability of success for Ctexli did not alter their price target, reflecting belief in the drug’s clinical data and market potential.
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