FDA approval summary for lonafarnib (Zokinvy) for the treatment of Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies.

The U.S. Food and Drug Administration recently approved lonafarnib as the first treatment for Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies. This approval was primarily based on a comparison of patients with HGPS treated with lonafarnib in 2 open-label trials with an untreated patient cohort. With up to 11 years of follow-up, it was found that the lonafarnib treated patients with HGPS had a survival benefit of 2.5 years compared with the untreated patients with HGPS. This large treatment effect on the objective endpoint of mortality using a well-matched comparator group mitigated potential sources of bias and together with other evidence, established compelling evidence of a drug effect with benefits that outweighed the risks. This approval is an example of U.S. Food and Drug Administration's regulatory flexibility for a rare disease while ensuring that standards for drug approval are met.

Content analysis of nutritional information in paediatric oral health education leaflets.

BACKGROUND: The aim of this study was to determine if paediatric oral health education leaflets with a food and nutritional focus provide messages that are clear and consistent with the current Australian Dietary Guidelines and the Infant Feeding Guidelines. METHODS: Forty-three leaflets aimed at parents were sourced from Australian state and territory Health Departments, oral health industry partners and commercial organisations, and a content analysis was performed. Recommendations on food and drink type, consumption frequency and general diet and nutrition advice were considered and cross-referenced with the Australian Dietary Guidelines and the Infant Feeding Guidelines to identify areas of consistency and discrepancy. RESULTS: Twenty leaflets recommended reducing the consumption of sugary and/or acidic food, while 23 leaflets recommended reducing the consumption of sugary and/or acidic drinks. The majority of the leaflets advised water (n = 35) and milk (n = 23) to drink. Although 33 leaflets encouraged a healthy diet, seven of these did not specify what a healthy diet was. Twenty-eight leaflets provided early childhood-related (0-2 years) feeding advice. Confusing messages were found in nine leaflets, with ambiguous recommendations that were open to individual interpretation. CONCLUSIONS: There were some inconsistencies between the leaflets and the dietary and infant feeding guidelines in Australia; and across the leaflets, as not all important messages were included in any one leaflet. Government Health Departments and other relevant agencies should ensure that advisory messages regarding diet, particularly those with dental implications, are clear, complete and consistent across all dental educational leaflets.