FOSTER CITY, Calif. - Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) received a positive opinion from the European Committee for Medicinal Products for Human Use (CHMP) for its drug LIVMARLI® (maralixibat) oral solution for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC), a rare genetic liver disease. This recommendation is a critical step towards potential marketing authorization in the European Union.
The CHMP's endorsement, announced today, is based on data from the Phase 3 MARCH study, which showed a highly statistically significant reduction in the severity of pruritus, a common symptom of PFIC, compared to a placebo. The study included 93 patients across various genetic types of PFIC, making it the largest randomized trial conducted for this condition.
In addition to the CHMP's positive opinion, the Committee for Orphan Medicinal Products (COMP) has also recommended maintaining the Orphan Drug Designation for LIVMARLI in PFIC. The European Commission is expected to make a final decision on the marketing authorization for LIVMARLI in the third quarter of 2024.
Chris Peetz, CEO of Mirum Pharmaceuticals, expressed optimism about the potential approval, highlighting the significant improvement in key liver health parameters that LIVMARLI could provide for PFIC patients starting from three months of age.
LIVMARLI is currently approved in Europe for treating cholestatic pruritus in patients with Alagille syndrome (ALGS) aged two months and older. In the United States, the drug is approved for the same condition in ALGS patients three months of age and older and for cholestatic pruritus in PFIC patients five years of age and older. Mirum is also planning to introduce a higher concentration formulation of LIVMARLI in the United States to expand its label for younger PFIC patients later this year.
PFIC leads to progressive liver disease, often resulting in liver failure. Patients with PFIC experience severe itching, jaundice, growth failure, and an increasing inability of the liver to function. The disease is estimated to affect one in every 50,000 to 100,000 births in the United States and Europe.
The safety information for LIVMARLI includes warnings about potential liver injury and gastrointestinal problems, and the drug is not recommended for PFIC type 2 patients with a severe defect in the bile salt export pump protein.
This report is based on a press release statement from Mirum Pharmaceuticals.
InvestingPro Insights
As Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) awaits the European Commission's final decision on the marketing authorization for LIVMARLI®, the company's financial health and market performance provide additional context for investors. According to InvestingPro data, Mirum boasts a significant revenue growth of 133.9% over the last twelve months as of Q1 2024, signaling strong sales and potentially increased market penetration. This is further supported by a gross profit margin of 73.26%, indicating the company's ability to maintain profitability with its current cost structure.
Despite the absence of dividends and profitability over the last twelve months, an InvestingPro Tip suggests that Mirum's liquid assets exceed its short-term obligations, providing the company with a solid liquidity position to manage its operations and investments. Moreover, the company is operating with a moderate level of debt, which could offer some financial flexibility as it continues to expand its product offerings and market reach.
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