Saturday, June 15, 2024

Breakthrough in Hepatitis Treatment: FDA Grants Breakthrough Therapy Designation to DURECT’s Larsucosterol

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Hepatitis treatment advancements have been made as DURECT Corporation announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to larsucosterol, a medication designed to treat patients with severe alcohol-associated hepatitis (AH). This designation is based on positive results from the Phase IIb AHFIRM trial, which demonstrated promising safety and efficacy outcomes. Initial data was announced last year, with additional clinical data expected to be presented at the European Association for the Study of the Liver (EASL) Congress next month.

“We’re pleased with the FDA’s decision to grant Breakthrough Therapy designation to larsucosterol, as it further recognizes its potential to save the lives of AH patients,” said James E. Brown, DVM, President, and CEO of DURECT, in a press release. “AH has a high mortality rate and no currently approved treatments, so there is a great need for a safe and effective therapy.”

The Alcoholic Hepatitis: Evaluation of Safety and Efficacy of Larsucosterol in Multinational Phase IIb Randomized Study (AHFIRM) Phase IIb trial was a randomized, double-blind, placebo-controlled, international, multi-center study aimed at evaluating the safety and efficacy of larsucosterol in subjects with AH. The trial enrolled 307 patients across three arms, focusing on the primary outcome measure of the 90-day incidence of mortality or liver transplantation for patients treated with larsucosterol compared to those treated with a placebo. The secondary endpoint was 90-day survival.

Larsucosterol Shows Promising Results in Reducing Mortality for Alcohol-Associated Hepatitis

Data released in November indicated that larsucosterol doses of 30 mg and 90 mg demonstrated a noteworthy trend in reducing mortality at 90 days. However, the numerical improvement in mortality or transplantation at 90 days did not achieve statistical significance. Approximately 76% of patients enrolled in the trial exhibited a more distinct reduction in mortality compared to the placebo group.

“The topline results from AHFIRM provide compelling evidence that administration of larsucosterol can reduce mortality at 90 days in this devastating disease,” said Brown. “We have a strong rationale to advance larsucosterol into a Phase III registration trial designed with adequate power to detect a statistically significant result using 90-day mortality as the primary endpoint. We look forward to meeting with the FDA to discuss the next steps. Based on the strength of the clinical data generated to date, if approved, larsucosterol could save many patients’ lives. We extend our thanks to all the patients, families, clinical trial investigators, and staff across the multiple sites globally who have worked with the DURECT team to bring larsucosterol to this advanced stage.”

Alcohol-associated hepatitis is a severe condition characterized by inflammation of the liver due to excessive alcohol consumption. Common symptoms include abdominal tenderness, nausea, weight loss, poor appetite, and vomiting blood or material resembling coffee grounds. According to Hopkins Medicine, AH can be diagnosed through various methods such as blood tests, abdominal ultrasounds, CT scans, MRI, and liver biopsies.


FDA Grants Breakthrough Therapy Designation to Larsucosterol for Severe Alcohol-Associated Hepatitis

The Mayo Clinic notes that AH typically occurs in individuals who drink heavily over many years. However, the link between drinking and AH is not straightforward; not all heavy drinkers develop the condition, and some individuals who drink much less may still get the disease. The FDA’s Breakthrough Therapy Designation for larsucosterol is a critical step toward addressing the unmet medical need for an effective treatment for severe alcohol-associated hepatitis (AH). With no currently approved treatments, this designation underscores the urgency and importance of finding a viable therapy for this condition, which is associated with a high mortality rate.

The development and potential approval of larsucosterol could offer a life-saving option for many patients suffering from severe AH. As a promising new treatment, larsucosterol has the potential to significantly improve patient outcomes and provide hope for those affected by this devastating disease. The forthcoming Phase III trial, informed by the promising results of the Phase IIb AHFIRM trial and recent FDA feedback, will be crucial in further establishing the efficacy and safety of larsucosterol. Successful results from this trial could lead to the drug’s approval and availability, providing a much-needed therapeutic option for patients suffering from severe AH.

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In conclusion, the FDA’s recognition of larsucosterol as a Breakthrough Therapy underscores the potential impact of this medication in treating severe alcohol-associated hepatitis. As DURECT Corporation advances its clinical development, the medical community and patients alike remain hopeful for a new, effective treatment that can significantly improve patient outcomes and save lives.


Resource: DURECT Corporation, May 22, 2024

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