Global Phenylketonuria Treatment Market Size (2024 - 2029)

Phenylketonuria Treatment Market Analysis

The phenylketonuria treatment market is expected to register a CAGR of 11% over the forecast period (2022-2027).

The COVID-19 pandemic has moderately impacted healthcare systems and the market. Neurocognitive outcome and phenylketonuria (PKU) prognosis are directly related to lifelong phenylalanine (Phe) levels and treatment adherence. For instance, In june 2021, as per the national library of medicine data, PKU patients had to adjust their dietary restrictions to the new environment. While younger patients may have been less exposed (meals strictly according to diet plan regardless of setting), adolescent and adult patients strongly reflected the obligation to stay at home by showing better metabolic control. Multiple factors could have contributed to this, including the availability of teleconsultancy, which allowed for easier connections. The similar source stated that how the pandemic and the environment can significantly impact PKU treatment adherence and how removing distance barriers can improve and optimize metabolic compliance. For instance, In June 2021, As per the data published by Europe PMC, the COVID19 pandemic strongly impacted people's life with lifestyle habits changing consistently. PKU patients had to adapt their dietary restrictions to the new environment they were exposed to and, if younger patients could have been less exposed (meals strictly according to diet plan independently from setting adolescentsent and adults strongly reflected the obligation to stay home by showing better metabolic control. Multiple factors could have played a role in that and the availability of teleconsultancy may have contributed allowing easier connections, but data demonstrate how the pandemic and the environment can strongly impact PKUs adherence to treatment and how removing distance barriers can ameliorate and optimize metabolic compliance. Thus, the abovementioned factors shows how the market has been impacted during a pandemic and it is expected to gain traction quickly with the resumption of economic activities.

Increasing clinical trials & research development activities are further boosting the growth of the market. For instance, In August 2022, BioMarin Pharmaceutical Inc. conducted a Phase 1/2 Open-Label, Dose Escalation research in Phenylketonuria patients with Plasma Phe Levels > 600 mol/L to investigate the safety and efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase. Moreover, PTC Therapeutics also conducted a clinical trial to assess the efficacy of PTC923 in lowering blood phenylalanine (Phe) levels in Phenylketonuria patients, as evaluated by the mean change in blood Phe levels from baseline to Weeks 5 and 6. (that is, the average of each respective treatment dose 2-week period of double-blind treatment).

Furthermore, Rising PKU awareness programs provided by local governments, national governmental organizations, and regulatory bodies and healthcare system assistance in developed and developing regions are driving factors for phenylketonuria treatment. Increasing awareness about PKU and medical food use is expected to boost demand for phenylketonuria treatment in the future. For instance, the National Phenylketonuria Alliance is a collaboration of PKU community members joining together as a national voice and supporting local efforts to raise PKU awareness and educate PKU patients, while ultimately looking for a cure. This alliance works to spread awareness through digital and direct interaction channels regarding the quality of life. Thus, the market is expected to drive in the forecast period from the abovementioned factors. But the high cost of treatment can hamper the growth of the market during the forecast period.

Phenylketonuria Treatment Industry Segmentation

Phenylketonuria is a birth defect, which occurs due to the mutation of the gene that is responsible for encoding phenylalanine hydroxylase. The presence of phenylalanine is higher in blood levels in the affected people. The phenylketonuria treatment market is segmented by Drugs (Kuvan, Playnziq, and Other Drugs) and Geography (North America, Europe, Asia-Pacific, and Rest of the World). The report offers the value (USD million) for the above segments.