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FDA Updates for the Week of Jan. 16, 2023


This week the FDA approved new indications for already available therapies, including Brukinsa for chronic lymphocytic leukemia and an accelerated approval for Tukysa for HER2-positive colorectal cancer, as well as an extended-release form of risperidone for schizophrenia. The agency also issued a complete response letter for Lilly's Alzheimer’s drug and accepted an application for a NASH drug.

FDA approves Brukinsa for chronic lymphocytic leukemia.

he FDA has approved Brukinsa (zanubrutinib) to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). Developed by BeiGene, Brukinsa is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK). BTK is an important enzyme in the B-cell receptor signaling pathway, regulating cell proliferation and cell survival in various B cell malignancies, including chronic lymphocytic leukemia.

Chronic lymphocytic leukemia is a slow growing type of B-cell malignancy, classified by overgrowth of white blood cells in the bone marrow. It is the most common leukemia in adults. It typically affects older white men with a median age at diagnosis of 70. In the United States, there will be an estimated 4,410 death this year due to chronic lymphocytic leukemia.

Brukinsa has a 30-day wholesale acquisition cost of $14,487. A company spokesperson said a patient support program can answer questions about treatment and provide insurance coverage assistance.

FDA approves Tukysa for HER2 positive colorectal cancer.

The FDA has granted an accelerated approval for Seagen’s Tukysa (tucatinib) for adult patients with unresectable or metastatic colorectal cancer. It is indicated in combination with trastuzumab as a second-line treatment for patients who are HER2-positive. Tukysa is an oral medicine that is a tyrosine kinase inhibitor of the HER2 protein.

Tukysa was granted accelerated approval based on tumor response rate and durability of response from the phase 2 MOUNTAINEER clinical trial. The results showed a 38% overall response rate in the patients who received the combination treatment. Complete responses were observed in 3.6% of patients, and partial responses were observed in 35% of patients. The median duration of response was 12.4 months.

FDA issues complete response for Lilly Alzheimer’s drug.

The FDA has issued a complete response letter for Eli Lilly’s accelerated approval submission of donanemab to treat patients with early Alzheimer’s disease. The agency indicated that there too few patients with at least 12 months of data provided in the submission.

In the complete response letter, the FDA specifically requested that Lilly provide data from at least 100 patients who received a minimum of 12 months of treatment with donanemab. The phase 2 trial had been designed to allow patients to complete their course of treatment when they reached a predefined level of amyloid plaque clearance. While the trial included more than 100 patients treated with donanemab, many patients were able to stop as early as six months. The FDA indicated that the data to meet the exposure expectation would likely need to include the unblinded controlled safety data from TRAILBLAZER-ALZ 2.

Lilly’s confirmatory phase 3 TRAILBLAZER-ALZ 2 trial is ongoing, with topline data expected in the second quarter of 2023. The company said this will form the basis of donanemab’s application for traditional approval.

FDA approves extended-release risperidone for schizophrenia.

The FDA has approved Rykindo (risperidone) to treat adults with schizophrenia and as adjunctive therapy to lithium or valproate for the maintenance treatment of bipolar I disorder in adults. Developed by China-based company Luye Pharma Group, Rykindo is an extended-release injectable suspension administered via intramuscular injection once every two weeks. It was developed using Luye Pharma’s microsphere technology platform.

According to the labeling, the effectiveness of Rykindo was established, in part, on the basis of the established effectiveness of the oral formulation of risperidone as well as in a 12-week, placebo-controlled trial in adult inpatients and outpatients. Efficacy data were obtained from 400 patients with schizophrenia who were randomized to receive injections of 25 mg, 50 mg, or 75 mg Rykindo or placebo every two weeks.

Rykindo has a black-box warning about use in elderly patients. Elderly patients with dementia-related psychosis treated with antipsychotic drugs are at an increased risk of death. It is not approved for use in patients with dementia-related psychosis.

FDA accepts application for NASH drug.

The FDA has accepted Intercept Pharmaceuticals’ resubmission of its new drug application (NDA) for obeticholic acid (OCA) seeking accelerated approval to treat patients with pre-cirrhotic liver fibrosis due to nonalcoholic steatohepatitis (NASH). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 22, 2023.

NASH is a serious progressive liver disease caused by excessive fat accumulation in the liver that induces chronic inflammation, resulting in progressive fibrosis that can lead to cirrhosis, eventual liver failure, cancer and death. Advanced fibrosis is associated with a substantially higher risk of liver-related morbidity and mortality. There are currently no medications approved for the treatment of NASH.

Intercept had resubmitted the NDA in December 2022, following the FDA’s complete response letter that was issued in June 2020. At issue was Intercept’s use of a surrogate endpoint. The agency wanted to see additional post-interim analysis efficacy and safety data from the REGENERATE study.

The new submission contains data from two interim 18-month analyses from the pivotal phase 3 REGENERATE study in patients with pre-cirrhotic liver fibrosis due to NASH. In these analyses, which were announced in July 2022, OCA 25 mg demonstrated double the response rate of placebo in reduction in liver fibrosis stage without worsening of any of the three histologic components of NASH.

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