CONSHOHOCKEN, PA — Madrigal Pharmaceuticals, Inc., a Montgomery County-based biopharmaceutical company, has received accelerated approval from the U.S. Food and Drug Administration (FDA) for Rezdiffra. This game-changing medication, used in conjunction with diet and exercise, targets noncirrhotic NASH—a serious liver condition—associated with moderate to advanced liver fibrosis.
For over 15 years, Madrigal’s small research team, led by founder Dr. Becky Taub, dedicated themselves to address one of the most daunting challenges in drug development. Their tireless efforts have resulted in the first FDA-approved therapy for NASH, formally labeled nonalcoholic steatohepatitis, a severe, progressive liver disease that, until now, remained without an approved remedy.
“The accelerated approval of Rezdiffra represents a historic moment for the NASH field,” said Bill Sibold, CEO of Madrigal. “This signifies the best of what our industry is capable of.”
Rezdiffra, an oral THR-β agonist designed to target key underlying causes of NASH, comes as a much-anticipated relief for patients who have been waiting years for a therapy. The new drug was sanctioned based on the Phase 3 MAESTRO-NASH trial results, which showcased significant improvement in patients after 52 weeks of treatment at both 100mg and 80mg doses.
Dr. Taub expressed her gratitude to the many patients who participated in the studies, “We believe Rezdiffra will alter the treatment paradigm for NASH sufferers, providing them with a therapy directly targeted at their liver condition to help improve fibrosis and resolve NASH before they progress to cirrhosis.”
This breakthrough is expected to bolster efforts in the NASH community, “accelerating our attempts to improve disease education, build care pathways, and expand investment in NASH research,” remarkably claims Wayne Eskridge, CEO of the Fatty Liver Foundation.
Rezdiffra’s approval marks a fundamental shift in NASH treatment. Interestingly, the prescribed information doesn’t require a liver biopsy for diagnosis. The recommended daily dosage is weight-based, with 80mg for patients weighing less than 100kg and 100mg for patients weighing more.
Dr. Stephen Harrison, Chairman of both Pinnacle Clinical Research and Summit Clinical Research, sees Rezdiffra as “a true game-changer for healthcare providers, the research community, and above all, patients living with this serious liver condition.”
The MAESTRO-NASH outcomes study remains ongoing, designed to generate confirmatory data that is expected to verify clinical benefits and potentially support full approval.
Rezdiffra is projected to be available to U.S. patients in April and will be distributed through a limited specialty pharmacy network. Madrigal has committed to helping relevant patients access Rezdiffra through the Madrigal Patient Support Program, a program designed to assist patients navigate insurance and affordability issues.
While the celebrations are in full swing for the NASH community, researchers remain on high alert. The ultimate goal now is to assess whether the positive outcomes observed in the MAESTRO studies will lead to a reduced risk of progression to cirrhosis, liver failure, need for liver transplant, and premature mortality. For all intents and purposes, the fight against NASH is far from over, but Rezdiffra’s arrival is undoubtedly a significant step forward.
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