In what may be a moot exercise for now, the Institute for Clinical and Economic Review (ICER) found that the long-term effects of Intercept Pharmaceuticals Inc.’s obeticholic acid (OCA) on the quality of life and health of patients with fibrosis due to nonalcoholic steatohepatitis (NASH) are uncertain. The New York-based company had been seeking accelerated approval of OCA for use in NASH. Instead, the FDA delivered a complete response letter (CRL) last month. As a result, ICER canceled its August meeting that had been scheduled to review its evidence report for OCA. But it still released the report, which found that the evidence for use of the drug in NASH with F2 fibrosis is insufficient, while it is “promising but inconclusive” in F3 fibrosis. Potential cardiovascular risks of OCA led to uncertainties about whether it would benefit or harm many patients, ICER said. In addition, the report found that the evidence of using a generic diabetes drug, pioglitazone, to treat NASH also is promising but inconclusive. Given Intercept’s CRL, ICER said it wouldn’t issue a health-benefit price benchmark for OCA at this time. However, it said that if OCA as a treatment for NASH were priced the same as Ocaliva, Intercept’s OCA product for biliary cholangitis, it “would far exceed all commonly cited thresholds for cost-effectiveness.”
The SEC agreed to settlements with two cousins, Edmond Leung and Joseph Zhang, to resolve charges of insider trading involving Sangamo Therapeutics Inc., of Brisbane, Calif. According to the SEC, Leung was a former manager in Sangamo’s information technology department. When he learned in August 2016 that the company was delaying clinical trials of gene therapies it was developing to treat mucopolysaccharidosis type I and II, he sold 1,439 shares of Sangamo stock before the delay was announced, avoiding $2,863 in losses. Then in May 2017, Leung learned of a pending collaboration, valued at more than $500 million, between Sangamo and Pfizer Inc. He gave the information to his cousin, who immediately purchased 16,900 shares of Sangamo stock hours before the collaboration was announced. “In the days following the announcement, Zhang sold his entire position in Sangamo stock for a $66,703 profit,” according to the SEC. Under his settlement with the SEC, Leung will disgorge $2,863 plus $493 in interest and pay a civil penalty of $69,566. In a separate settlement, Zhang agreed to disgorge $66,703 plus $5,573 in interest and pay a civil penalty of $66,703.
The FDA said it is now supporting the current version of the International Institute of Electrical and Electronics Engineers’ Biocompute standard for use in regulatory submissions and is including the standard as an update in the FDA Data Standards Catalog for the submission of high-throughput sequencing (HTS) data in drug and biologic applications. The Biocompute standard will facilitate the exchange of HTS bioinformatics workflows by specifying the information needed to understand and organize bioinformatic analyses, the agency said in a notice published in Wednesday’s Federal Register.
The EMA said Tuesday that it has set up an infrastructure to support the monitoring of the efficacy and safety of COVID-19 treatments and vaccines used in day-to-day clinical practice. The infrastructure is underpinned by three contracts for observational research that the EMA signed with academic and private partners over recent months. The goal is to be able to effectively monitor vaccines in the real world as soon as they are authorized and to support the safe and effective use of COVID-19 vaccines and drugs.
Although Stason Pharmaceuticals Inc., of Irvine, Calif., recalled a lot of temozolomide capsules that failed dissolution testing after the specification standard was expanded, the company failed to determine a clear root cause of the failure, the FDA said in a warning letter posted this week. The company also failed to provide adequate details regarding a new desiccant study it was initiating and the formulation modification being evaluated. In addition, it didn't provide the study protocol or its new corrective action and preventive action to avoid future dissolution failures. Based on the company’s response to citations stemming from an October 2019 inspection, the letter said Stason lacked adequate interim measures to address cleaning issues, including a verification failure, following the manufacture of methotrexate tablets. It also noted that a demonstration performed during the inspection revealed that a computer operating a spectrophotometer system used for finished product release and stability testing for several drugs wasn't secured, so data files could be deleted without the knowledge of the quality unit. At the time of the inspection, the company acknowledged that its software wasn't working as intended and that it lacked the knowledge or experience to troubleshoot the issue, according to the letter.
Signa SA de CV received an FDA warning letter resulting from a December inspection of its drug manufacturing facility in Toluca, Mexico. The letter cited the company’s failure to adequately investigate out-of-specification (OOS) results for active pharmaceutical ingredients (APIs) and to implement appropriate corrective actions. “Notably, your firm has experienced several additional failures during the production history of this API," the FDA said. For instance, it noted after obtaining an OOS assay result during the release testing of an API batch in October 2018, the facility obtained passing retest results and invalidated the original OOS result. “Your firm indicated that a likely root cause was instability of the analytical balance due to the presence of too many analysts in the weighing room,” the letter said. “There was no evidence that the presence of multiple analysts in the room affected the sensitivity of the analytical balance and therefore contributed to the OOS results.”