Rare Disease Report

Survey Results Indicate PKU Patients' Lack of Adherence to Supplements

MARCH 12, 2018
Mathew Shanley
At the 40th annual meeting of the Society for Inherited Metabolic Diseases (SIMD) in San Diego this weekend, results from a global market research survey on phenylketonuria were presented, and concluded that patients’ adherence to currently prescribed supplements is a significant issue.

The data were presented by APR Applied Pharma Research s.a., and included information provided by 96 interviews with pediatricians, dieticians, PKU caregivers, and PKU patients. The qualitative research was conducted by IDR Medical in the United States and five European countries, and worked to provide insights into the current landscape of PKU management, including patient flows, stakeholders’ influence and decision criteria for medical food adoption.

PKU is a genetic disorder characterized by a patient’s lack of ability to metabolize the amino acid (AA) phenylalanine (Phe). The survey results presented at the SIMD meeting indicate that patients’ adherence to the currently prescribed AA supplements is primarily driven by taste fatigue. Quality long-term patient compliance to the recommended dietary regimen has yet to be achieved, and is critical to attaining the required balance between sufficient nutritional intake and good metabolic control with Phe levels within the ranges.

“We are very pleased of the results of this extensive research, now validated by the poster Phenylketonuria: not all problems are solved, as they confirm product palatability as one of the main open issues in the management of PKU, resulting in poor long-term compliance,” said Paolo Galfetti, CEO of Applied Pharma Research in a press release. “We are confident that APR’s innovative Medical Food will deliver tangible and significant benefits to PKU patients, by making easier to accept life-long daily dietary restrictions and thus contributing to their wellbeing.”

In the survey, PKU patients ranked taste as the top priority when choosing an AA supplement, with good control of Phe levels and the contribution to personal wellbeing as the second and third most important aspects. The survey results support previous data and the relevance of an innovative supplementation that would address these needs, and thus significantly contribute to revolutionizing PKU management.

APR has developed a patented new technology, known as Physiomimic Technology. It was designed with the intention of offering an innovative Phe-free AA supplement to PKU patients, while also improving their quality of life (QOL). It is expected to concurrently help patients endure the prescription of supplementation.

In short, the technology is expected to mask the naturally unlikable taste and odor of some amino acids. It has the potential to offer patients an appetizing medical food, while also helping them obtain a satisfying and enduring compliance to the dietary treatment.

The company plans to present further results and advancements at the 56th Annual Symposium of the Society for the Study of Inborn Errors of Metabolism in Athens, Greece in September.

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