Mirum Pharmaceuticals

Ipsen's Elafibranor Shows Promise in Phase II Trial for Primary Sclerosing Cholangitis

a month ago

• Elafibranor demonstrated a favorable safety profile and significant dose-dependent efficacy in the Phase II ELMWOOD trial for primary sclerosing cholangitis (PSC), a rare liver disease with no currently approved treatments. • Patients treated with elafibranor showed significant improvements in liver biochemical parameters, including alkaline phosphatase, with stabilization of non-invasive markers of liver fibrosis compared to placebo. • The 120mg dose of elafibranor significantly improved pruritus symptoms, offering potential relief for a common and distressing symptom experienced by PSC patients.

FDA Approves Tablet Formulation of LIVMARLI for Rare Pediatric Liver Diseases

2 months ago

• Mirum Pharmaceuticals' LIVMARLI (maralixibat) has received FDA approval for a new tablet formulation to treat cholestatic pruritus in patients with Alagille syndrome and Progressive Familial Intrahepatic Cholestasis. • The tablet version provides a convenient one-tablet per dose option for older patients, while maintaining the liquid formulation for younger patients, enhancing treatment flexibility for physicians and patients. • LIVMARLI, an ileal bile acid transporter (IBAT) inhibitor, is now approved in over 40 countries for ALGS and 30 countries for PFIC, with the new tablet formulation expected to be available in June 2025.

Dupilumab Shows Strong Efficacy in Chronic Spontaneous Urticaria Across All BMI Ranges

3 months ago

• Phase 3 LIBERTY-CSU CUPID trials demonstrate dupilumab's effectiveness in reducing itch and urticaria activity in patients unresponsive to H1-antihistamines. • Clinical benefits of dupilumab were consistent across all BMI ranges, including patients in obese categories, providing important efficacy data for diverse patient populations. • FDA review of dupilumab for chronic spontaneous urticaria is underway with a target action date of April 18, 2025, potentially offering a new treatment option for resistant cases.

FDA Approves Genentech's TNKase for Acute Ischemic Stroke with Simplified Administration

3 months ago

• The FDA has approved Genentech's TNKase, a clot-dissolving agent, for treating acute ischemic stroke in adults, marking the company's second stroke treatment approval. • TNKase offers a streamlined five-second intravenous bolus administration, presenting a significant advantage over the current standard treatment Activase's 60-minute infusion protocol. • The approval is supported by a multi-center non-inferiority study across 22 Canadian stroke centers, demonstrating comparable safety and efficacy to Activase in patients with disabling neurological deficits.

FDA Approves Five Groundbreaking Treatments for Ultra-Rare Diseases in Recent Months

3 months ago

• The FDA has approved novel therapies for Duchenne Muscular Dystrophy, including ITF Therapeutics' Duvyzat (givinostat), which targets histone deacetylases to potentially slow muscle deterioration. • Breakthrough gene therapy Lenmeldy received approval for Metachromatic Leukodystrophy, offering the first targeted treatment option with significant survival benefits for pediatric patients. • Multiple rare diseases saw first-ever treatments, including Xolremdi for WHIM syndrome, dual approvals for Niemann-Pick Disease Type C, and Ctexli for Cerebrotendinous Xanthomatosis.

FDA Approves First Treatment for Cerebrotendinous Xanthomatosis: Mirum's Chenodiol Tablets

3 months ago

• The FDA has granted historic approval to chenodiol tablets (Ctexli) as the first-ever treatment for cerebrotendinous xanthomatosis, a rare genetic lipid storage disease affecting multiple organ systems. • Phase 3 RESTORE study demonstrated chenodiol's efficacy with a statistically significant 20-fold reduction in bile alcohols compared to placebo (P<0.0001) in CTX patients. • The treatment, administered as 250mg tablets three times daily, comes with monitoring requirements for liver toxicity and showed common side effects including diarrhea and headache.

FDA Approves ORLYNVAH™: First New Oral Treatment for Uncomplicated UTIs in 25 Years

4 months ago

• Iterum Therapeutics receives FDA approval for ORLYNVAH™, marking the first branded uncomplicated UTI treatment to enter the U.S. market in over 25 years and the first oral penem antibiotic approved in the country. • The drug demonstrates significant potential against multi-drug resistant pathogens, specifically targeting Escherichia coli, Klebsiella pneumoniae, and Proteus mirabilis in adult women with limited treatment options. • Iterum reports $24.1 million in cash reserves as of December 2024, with funding projected to sustain operations into the second half of 2025 while pursuing pre-commercialization activities.

Livmarli Shows Long-Term Efficacy in Relieving Itch for Adolescents with Alagille Syndrome

4 months ago

A new analysis of clinical trial data demonstrates that Livmarli (maralixibat) effectively reduces itching in adolescents with Alagille syndrome, with benefits maintained into late adolescence or adulthood. The study, funded by Mirum Pharmaceuticals, showed significant reductions in both itch severity scores and blood bile acid levels among patients treated before age 16.

Liver Cirrhosis Clinical Trial Pipeline Gains Momentum

4 months ago

The liver cirrhosis treatment market is experiencing significant growth, driven by advancements in antiviral therapies and regenerative medicine. Over 30 companies are actively developing new treatments, with several promising therapies currently in clinical trials.

Phase 3 Clinical Trial Enrolling to Test Maralixibat for Cholestasis-Related Itch

4 months ago

A Phase 3 clinical trial, EXPAND, is currently enrolling participants to evaluate the effectiveness of maralixibat in treating treatment-resistant itching associated with rare cholestatic conditions. The trial aims to enroll up to 90 participants aged 6 months and older across multiple global sites, with results expected by 2026.

GSK's Linerixibat Shows Positive Phase III Results for Cholestatic Pruritus in Primary Biliary Cholangitis

6 months ago

• GSK's linerixibat met its primary endpoint in the GLISTEN Phase III trial, demonstrating a statistically significant reduction in itch for PBC patients with moderate to severe pruritus. • The trial evaluated linerixibat in PBC patients already receiving guideline-suggested therapies, treatment-naïve patients, and previously treated patients, showing potential as a targeted therapy. • Linerixibat, an ileal bile acid transporter (IBAT) inhibitor, could be the first global therapy specifically developed to treat itch in PBC, addressing a significant unmet need. • Preliminary safety results were consistent with prior studies, and full results from the GLISTEN trial will be presented at a future scientific congress.

Novartis and Apellis Advance C3G Therapies Towards FDA Submission

6 months ago

• Novartis' Fabhalta (iptacopan) demonstrated significant proteinuria reduction in the Phase III APPEAR-C3G trial, meeting its primary endpoint and supporting regulatory submissions. • Apellis' Empaveli (pegcetacoplan) showed a substantial reduction in proteinuria and C3c deposit clearance in the Phase III VALIANT trial, indicating early efficacy in C3G patients. • Nephrologists express a strong interest in new C3G therapies that can slow eGFR decline and reduce proteinuria, addressing the urgent need for innovative treatments. • Spherix data suggests treatment preferences may be influenced by administration route, with Fabhalta's oral administration potentially offering an advantage.

Volixibat Receives FDA Breakthrough Therapy Designation for Cholestatic Pruritus in Primary Biliary Cholangitis

8 months ago

• The FDA granted Breakthrough Therapy Designation to volixibat for treating cholestatic pruritus in patients with primary biliary cholangitis (PBC). • This designation is based on positive interim results from the Phase 2b VANTAGE study, showcasing statistically significant pruritus improvement versus placebo. • Volixibat, an oral ileal bile acid transporter (IBAT) inhibitor, could expedite development and review for this burdensome symptom of PBC. • Enrollment in the confirmatory portion of the VANTAGE study is ongoing, with completion expected in 2026, marking a significant step forward.