Intercept Pharmaceuticals, Inc. ICPT announced that the FDA’s Gastrointestinal Drugs Advisory Committee (GIDAC) Meeting has voted against the approval of the company’s new drug application (NDA) for obeticholic acid (OCA) for the treatment of pre-cirrhotic fibrosis due to non-alcoholic steatohepatitis (NASH).
NASH is a serious progressive liver disease caused by excessive fat accumulation in the liver that induces chronic inflammation, resulting in progressive fibrosis (scarring) that can lead to cirrhosis, eventual liver failure, cancer and death.
Twelve of the sixteen voting-eligible advisors voted negative on question, “given the available efficacy and safety data, do the benefits of OCA 25 mg outweigh the risks in NASH patients with stage 2 or 3 fibrosis?” Fifteen of 16 voting-eligible GIDAC members (with no abstentions) voted to “defer approval until clinical outcome data from trial 747-303 are submitted and reviewed, at which time the traditional approval pathway could be considered.”
The outcome of the meeting will provide insights to the FDA, which is scheduled to make a decision regarding the same on Jun 22, 2023. However, the FDA is not bound by this outcome.
Last week, shares of Intercept were down after the FDA publicly posted briefing documents in advance of this meeting due to concerns about drug-induced liver injury. The document stated that, during the review, the FDA identified modest benefits and serious risks of OCA for treating NASH.
The key issues for consideration in the benefit-risk assessment of OCA for NASH with fibrosis include the theoretical clinical benefits of a one-stage improvement in fibrosis, along with the risks of drug-induced liver injury, gallstone or bile duct stone/sludge and related complications, new-onset or worsening dyslipidemia, accelerating progression to developing prediabetes or diabetes and worsening of glycemic control in diabetic subjects.
Intercept previously faced roadblocks in getting its NDA accepted. The company received a complete response letter (“CRL”) from the FDA in 2020 stating that its NDA for OCA for the treatment of liver fibrosis due to NASH could not be approved in its present form.
The CRL indicated that the FDA determined that the predicted benefit of OCA, based on a surrogate histopathologic endpoint, remains uncertain and does not sufficiently outweigh the potential risks to support accelerated approval for treating patients with liver fibrosis due to NASH. This analysis was based on the data reviewed by the agency.
Intercept’s shares have lost 20.3% in the year so far compared with the industry’s 7.1% decline.
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While the NASH market promises potential with no approved therapies, it is challenging as well.
Several other companies are attempting to develop a treatment for the same condition.
Madrigal Pharmaceuticals, Inc. MDGL announced that its pipeline candidate resmetirom received Breakthrough Therapy designation from the FDA for treating patients with NASH with liver fibrosis. Madrigal is currently conducting four phase III clinical studies to demonstrate the safety and efficacy of resmetirom for the treatment of NASH: MAESTRO-NASH, MAESTRO-NAFLD-1, MAESTRO-NAFLD-OLE and MAESTRO-NASH-OUTCOMES.
Shares of Viking Therapeutics VKTX gained recently after the company announced positive top-line results from its phase IIb clinical study of VK2809 in patients with biopsy-confirmed NASH. The study achieved its primary endpoint, with patients receiving VK2809 experiencing statistically significant reductions in liver fat content from baseline to week 12 compared with placebo.
VK2809-treated patients demonstrated statistically significant reductions in low-density lipoprotein cholesterol, triglycerides and atherogenic lipoproteins compared with placebo. Viking Therapeutics plans to report 52-week biopsy data from the VOYAGE study in the first half of next year.
Intercept currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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