Phenylketonuria Treatment Market - Growth, Trends, COVID-19 Impact, and Forecasts (2022 - 2027)

Phenylketonuria Treatment Market - Growth, Trends, COVID-19 Impact, and Forecasts (2022 - 2027)

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.

Key Market TrendsKuvan Segment is Expected to Hold a Major Market Share in the Phenylketonuria Treatment Market

Kuvan is the only drug present currently for the efficient treatment of phenylketonuria, which is being manufactured by BioMarin Pharmaceuticals. It adds more BH4 compounds and helps in stimulating the phenylalanine hydroxylase enzyme, which then breakdown the phenylalanine protein. While the increasing launches and developments are further driving the growth of the market. For instance, In April 2021, Dr. Reddy’s Laboratories launched the Sapropterin Dihydrochloride Powder for Oral Solution, 100 mg), a therapeutic equivalent generic version of Kuvan (sapropterin dihydrochloride) Powder for Oral Solution, 100 mg, USP, approved by the US Food and Drug Administration (USFDA). For instance, In Aprail 2021, Dr. Reddy’s Laboratories has unveiled Sapropterin Dihydrochloride Powder for Oral Solution, 100 mg, a therapeutic equivalent generic version of Kuvan (sapropterin dihydrochloride) Powder for Oral Solution, 100 mg, USP, in the United States.

While the studies revealed the benefits associated with phenylketonuria are further propelling the growth of the market. As per the European public assessment report (EPAR) updated in September 2021, In Phenylketonuria treatment, the main study compared the reductions in blood phenylalanine in 88 patients treated with either Kuvan or placebo (a dummy treatment). Two other studies in 101 patients looked at how effective Kuvan was at allowing the patients to consume foods containing phenylalanine while keeping blood phenylalanine at target levels (i.e. phenylalanine tolerance). Kuvan was more effective than placebo at reducing blood phenylalanine levels in patients with PKU, achieving a reduction of 236 micromoles per liter after 6 weeks compared with an increase of 3 micromoles per liter seen with placebo. For instance, In Aprail 2021, Dr. Reddy’s Laboratories has unveiled Sapropterin Dihydrochloride Powder for Oral Solution, 100 mg, a therapeutic equivalent generic version of Kuvan (sapropterin dihydrochloride) Powder for Oral Solution, 100 mg, USP, in the United States.

In addition, Kuvan allowed patients with PKU who were not on a restricted diet to increase their daily phenylalanine intake by 17.5 mg per kg body weight after 10 weeks compared with 3.3 mg/kg with placebo. When Kuvan plus diet restriction was compared with diet restriction alone, the average daily phenylalanine intake that was tolerated after 26 weeks was 81 mg/kg in the Kuvan group and 50 mg/kg in the group on diet restriction alone. For the treatment of BH4 deficiency, which is a very rare condition, In August 2021, the national library of medicine presented the results of three studies from the published literature in which some patients were treated with sapropterin for an average of 15.5 months. In these studies, patients showed an improvement in blood phenylalanine levels and other markers of the disease when they were taking the medicine. Thus, the benefits associated with Kuvan are increasing the demand for Phenylketonuria Kuvan which is driving the growth of the market.

North America is Expected to Hold a Significant Share in the Market during the Forecast Period

North Ameriis ca expected to holdsignificantjor market share in the global phenylketonuria treatment market due to a higher incidence rate of phenylketonuria and the developmeof in treatmetypesype. For instance, In June 2022, as per the data by the national library of medicine, The overall incidence of phenylketonuria in the United States is about 1/15,000. This incidence is greater for Caucasian and Native American populations and less for African American, Hispanic and Asian populations. The same source also stated that In the United States, PKU is usually detected at birth by newborn screening tests and dietary therapy started in consultation with a dietitian and geneticist/metabolism specialist. Mild forms of PKU in a newborn can, however, go undetected if the mother is discharged too soon or if the newborn has not consumed any protein. Thus, the increasing incidence of Phenylketonuria are driving the growth for the phenylketonuria treatment market.

Growing research activities to treat Phenylketonurplayays a vital role. Moreover, governmental support for ongoing clinical research has allowed the United States to dominate the phenylketonuria treatment landscape. For instance, in May 2020, Retrophin, Inc. offered initial funds to the Rare Caregiver Respite Program. The program was launched by the National Organization for Rare Disorders (NORD), which aims to provide various services and financial assistance to the patients.

The presence of some prominent players such as Prinova Group LLC., Abbott Laboratories, Aegle Nutrition, Nova Care, ABCO Laboratories, Inc., Archer Daniels Midland Company, and other North American companies has created remarkable returns for the region. In December 2021, According to the National Organization of Rare Disorders (NORD) updated data, the incidence of Phenylketonuria from newborn screening programs ranges from one in 13,500 to 19,000 newborns in theUnited States. Phenylketonuria affects people from most ethnic backgrounds, although it is rare in Americans of African descent and Jews of Ashkenazi ancestry. Thus, the increasing cases of phenylketonuria is driving the growth of the market.

Competitive Landscape

The phenylketonuria treatment market is consolidated and consists of a few major players. Some of the companies currently dominating the market are BioMarin Pharmaceuticals Inc., Censa Pharmaceutical, Synlogic Inc., Erytech Pharma SA, Codexis Inc., SOM Innovation Biotech SL, Homology Medicines Inc., and Ultragenyx (Dimension Therapeutics).

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