Some patients take vitamin E supplements to reduce inflammation. Others get off-label prescriptions for the diabetes drug Actos, which can reduce fibrosis and scarring but can also cause weight gain.
NASH being the progressive form of nonalcoholic fatty liver disease (NAFLD), affects more than 115 million people worldwide, and estimated around 357 million people will be affected by 2030. Because NASH symptoms are often not overt, NASH is often underdiagnosed and underreported. Therapies for NASH have been notoriously difficult to develop.
Inside the NASH Drug Boom: New Drugs for ‘Silent’ Liver Disease
As the medical community began to understand NASH, Synta Pharmaceuticals gave Madrigal the funding it needed to get resmetirom drug into a Phase 2 study. The drug in the Phase 3 clinical trial was shown to improve fibrosis and resolve NASH. This means that the disease is no longer progressing, though patients remain in therapy.
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The company plans to submit the drug for FDA approval as a treatment for noncirrhotic NASH with liver fibrosis in the first half of this year, and approval could come by early 2024.
The other drug Intercept Pharmaceuticals’ ICPT obeticholic acid, was submitted for review by the regulator as a treatment for patients with pre-cirrhotic liver fibrosis due to NASH. A late-stage trial showed that the drug can treat the fibrosis that is common in NASH patients.
In recent years, GenFit GNFT, NGM Biopharmaceuticals NGM, and Intercept all saw mid or late-stage clinical trials fail, and those failures are reflected in the stock performances of those companies.
DA had accepted the resubmission of its application for obeticholic acid for a NASH indication and has watched its shares jump 36% over the last period. The announcement came after the therapy suffered previous failures in clinical trials to treat fibrosis in NASH patients.
At least half a dozen other companies have drugs in mid or late-stage clinical trials, including Lilly, which is testing Mounjaro to see if it can improve NASH outcomes, with data expected later this year().
Beyond pricing, there are several unknown things about how the market for NASH drugs will play out. Having two different drugs come to market around the same time may help lower prices by creating competition, although the therapies are expected to get different labels from the FDA.
Severe itching, a side effect of obeticholic acid seen in some patients, may also limit interest among patients. And it’s unclear how health insurers and pharmacy benefit managers will cover the new therapies, if and when they are approved.
Drugs are easily available when the diseases are socially accepted. Let us hope that NASH falls into the category similar to Type 2 diabetes drugs, where insurance is important.
References:
- Sheka, Adam C et al. Nonalcoholic Steatohepatitis: A Review. JAMA vol. 323,12 (2020): 1175-1183.(https://jamanetwork.com/journals/jama/article-abstract/2763297)
- Vuppalanchi, Raj et al. Therapeutic pipeline in nonalcoholic steatohepatitis. Nature reviews. Gastroenterology & hepatology vol. 18,6 (2021): 373-392.(https://www.nature.com/articles/s41575-020-00408-y)
Source: Medindia
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