Med Check
Roche’s Autism Drug Gets Breakthrough Status From FDA
Roche announced in February that the U.S. Food and Drug Administration (FDA) has designated the company’s experimental autism drug balovaptan (RG7314) a breakthrough therapy.
Several studies suggest that balovaptan—a vasopressin 1a (V1a) receptor antagonist—may improve social interaction and communication in people with autism. There are currently no FDA-approved drugs for the core symptoms of autism such as problems with social interaction and engaging in repetitive behaviors.
According to a Roche statement, the FDA Breakthrough Therapy designation for balovaptan “was primarily based on efficacy findings in the VANILLA (Vasopressin ANtagonist to Improve sociaL communication in Autism) study”—a phase 2 trial of the medication in adults with autism spectrum disorder (ASD). Balovaptan is currently in phase 2 trials in children and adolescents with ASD.
FDA Advisory Panel Rejects Claims of Reduced Risk Tobacco Product
The FDA Tobacco Products Scientific Advisory Committee voted against most of the marketing statements proposed by Philip Morris International for their heat-not-burn cigarette device. Philip Morris had hoped to sell its new technology, known as iQOS, as a “reduced risk” tobacco product.
According to the company, the iQOS gently heats a stick of tobacco without burning it, which reduces the exposure to tar and other toxic compounds found in traditional cigarettes.
However, in a unanimous vote (with one abstention), the nine-member FDA committee voted against the company’s statements, noting that the accompanying research evidence did not show that the device reduces deadly diseases tied to smoking.
The committee did support the company’s claim that completely transitioning from cigarettes to iQOS reduces exposure to harmful chemicals. That claim was considered the least significant, however, as it does not purport a direct health benefit.
Takeda Halts Phase 3 Trial Of Diabetes Drug For Alzheimer’s Prevention
Takeda Pharmaceuticals and its partner Zinfandel Therapeutics have terminated a phase 3 study testing the diabetes drug pioglitazone as a treatment to delay the onset of Alzheimer’s disease (AD).
The TOMORROW study was investigating whether low-dose pioglitazone could delay the onset of cognitive impairment in people who were considered at high risk of developing AD because of their genetic profiles. The study, which was initiated in 2013 and had already enrolled over 3,500 participants, was intended to run until 2019. However, an interim data analysis showed the drug was not delaying the onset of cognitive impairment and stopped due to futility.
Emiliangelo Ratti, head of Takeda’s Neuroscience Therapeutic Division, said that the two companies will conduct additional analysis of the existing data, including the performance of a genetic-based biomarker risk assignment algorithm, “with the hope that this information may ultimately help in the global fight against AD.”
Cyclopropyl Fentanyl Temporarily Placed in Schedule I
The Drug Enforcement Administration (DEA) has temporarily designated the synthetic opioid cyclopropyl fentanyl and its related isomers, esters, ethers, and salts as a Schedule I controlled substance. Schedule I drugs are deemed to have a high potential for abuse and no medical value.
The order became effective on January 4 and will remain until January 4, 2020. During this time, all individuals who handle or propose to handle cyclopropyl fentanyl are subject to the increased regulatory controls and administrative, civil, and criminal sanctions applicable to Schedule I controlled substances.
The DEA noted that at least 115 overdose deaths involving this synthetic opioid have been confirmed, but the number is likely higher as standard immunoassays may not differentiate this fentanyl analogue from fentanyl.
Long-Term Lisdexamfetamine Does Not Lead to Cognitive Impairments in Youth
The long-term use of the stimulant lisdexamfetamine dimesylate (LXD; Vyvanase) does not appear to adversely affect the cognition of children and/or adolescents with attention-deficit/hyperactivity disorder (ADHD). These findings arose from a two-year open-label trial sponsored by Shire Pharmaceuticals, the manufacturer of Vyvanase, and were published in CNS Drugs.
In the study, 314 youth (aged 6 to 17 years) with ADHD received dose-optimized open-label LXD (30 mg/day, 50 mg/day, or 70 mg/day) for 104 weeks. Cognitive function was assessed at weeks 0 (baseline), 4, 24, 48, 72, and 104 using multiple tests from the Cambridge Neuropsychological Test Automated Battery (CANTAB).
No clinically significant reductions (5 percent or greater change) in any CANTAB scores were observed over the 104-week study period. Potentially meaningful improvements from baseline were observed at the end of the study for some of the assessments, such as the Spatial Working Memory and Stop Signal Task tests.
However, the investigators cautioned, “In the absence of a placebo control group, it is not possible to dissect drug-induced changes in performance in cognitive tasks from natural improvement over time or improvement owing to practice effects.” ■
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Psychiatrics News
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Published online: 1 March 2018
Published in print: February 17, 2018 – March 2, 2018
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