Chiesi Global Rare Diseases is committing up to $115 million to begin a collaboration on Arbor Biotechnologies’ in vivo gene-editing therapy for primary hyperoxaluria type 1, an inherited liver disorder. It’s the rare disease company’s first foray into genetic medicines.
Novo Nordisk is acquiring Akero Therapeutics for drug candidate efruxifermin, which executive say could be first and best in the emerging class of FGF21 analogs for the fatty liver disease MASH. The deal comes months after Novo’s internal FGF21 drug candidate failed a Phase 2 trial.
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Intercept Pharmaceuticals is voluntarily withdrawing from the market Ocaliva, a drug for the rare liver disease primary biliary cholangitis. While Ocaliva became an important new treatment option when it won accelerated FDA approval in 2016, PBC patients now have new FDA-approved medicines from Ipsen and Gilead Sciences that don’t pose the same safety risks.
Approval is only the beginning. Now comes the harder part: identifying the right patients, detecting the disease earlier, and monitoring outcomes so that this promising treatment is effective at scale.
Etiome’s precision medicine approach introduces a temporal component to treating disease — drugs developed specifically to treat disease at particular points in time even before symptoms show. The Flagship Pioneering startup’s approach offers the potential to prevent disease; in cases where disease is already present, Etiome aims to halt or even reverse it.
Akero Therapeutics reported that at 96 weeks, 39% of patients who received the 50 mg dose of efruxifermin achieved improvement in fibrosis, or liver scarring, caused by the fatty liver disease MASH. This study previously failed to achieve statistically significant improvement at 36 weeks, but Akero executives say the latest results show that longer dosing matters.
Healthcare in the U.S. today exists in a complex, fragmented and costly system, and the people this system is designed for deserve a holistic approach to best address their individual health needs.
Overcoming the systemic, structural and cultural issues that lead to poor nutrition is complex, making it even more essential for both private and public investment in food as health programs and policies that have been shown to be effective.
Novo Nordisk’s Wegovy achieved statistically significant improvement in a Phase 3 study in the fatty liver disease MASH. Based on these preliminary results, the company plans to seek U.S. and European Union approvals in this indication in 2025.
Livdelzi is now FDA-approved for treating the rare liver disease primary biliary cholangitis. Gilead Sciences added the drug to its pipeline via the $4.3 billion acquisition of CymaBay Therapeutics earlier this year.
Iqirvo failed as a treatment for the fatty liver disease MASH, but the drug is now FDA approved in primary biliary cholangitis. An Intercept Pharmaceuticals drug already treats this rare liver disease and Gilead Sciences is poised to compete with its PBC drug approaching an FDA decision this summer.
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The first FDA-approved MASH drug doesn’t treat patients with liver cirrhosis. A new Boehringer Ingelheim/Ochre Bio collaboration is researching regenerative medicines that could treat patients in this most advanced stage of the fatty liver disease.
Acquiring CymaBay Therapeutics gives Gilead Sciences another drug for its liver disease portfolio. CymaBay’s molecule, seladelpar, is under FDA review for treating primary biliary cholangitis, a rare disease affecting the bile ducts of the liver.
Sanofi is acquiring Inhibrx to get its hands on a drug candidate in pivotal testing for the protein deficiency alpha-1 antitrypsin deficiency, which leads to lung and liver problems. Inhibrx’s remaining assets will be spun out into a new company.
Intercept Pharmaceuticals’ acquisition by Alfasigma comes three months after the FDA again rejected the biotech’s drug as a treatment for the fatty liver disease NASH. But Intercept still has rare liver disease assets, and Italy-based Alfasigma says acquiring the company will help it expand in gastroenterology and hepatology.
The FDA again rejected Intercept Pharmaceuticals’ application seeking accelerated approval for its NASH drug and asked for more data. Instead, the biotech will stop all work in that fatty liver disease and focus on drugs for other serious but rare liver conditions.