The Phenylketonuria (PKU) drugs in development market research report provides comprehensive information on the therapeutics under development for Phenylketonuria (PKU), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. GlobalData’s report assesses key aspects of the companies and drugs in development for Phenylketonuria (PKU). Buy the report here.

The report also covers the descriptive pharmacological action of the therapeutics and the latest news and press releases. Additionally, the report provides an overview of the key players involved in therapeutic development for Phenylketonuria (PKU) and features dormant and discontinued products.

GlobalData tracks 35 drugs in development for Phenylketonuria (PKU) by 31 companies/universities/institutes. The top development phase for Phenylketonuria (PKU) is preclinical with 20 drugs in that stage. The Phenylketonuria (PKU) pipeline has 34 drugs in development by companies and one by universities/ institutes. Some of the companies in the Phenylketonuria (PKU) pipeline products market are: Moderna, Synlogic and Relief Therapeutics.

The key targets in the Phenylketonuria (PKU) pipeline products market include Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1), Phenylalanine Ammonia Lyase (Phenylalanine Deaminase or Phenylalanine Ammonium Lyase or PAL or EC 4.3.1.24), and Tetrahydrobiopterin (THB or BH4).

The key mechanisms of action in the Phenylketonuria (PKU) pipeline product include Phenylalanine 4 Hydroxylase (Phe 4 Monooxygenase or PAH or EC 1.14.16.1) Activator with 13 drugs in Phase II. The Phenylketonuria (PKU) pipeline products include two routes of administration with the top ROA being Oral and ten key molecule types in the Phenylketonuria (PKU) pipeline products market including Gene Therapy, and Small Molecule.

Phenylketonuria (PKU) overview

Phenylketonuria (PKU) is a rare inherited disorder that causes an amino acid called phenylalanine to build up in body. Symptoms include intellectual disability, psychiatric disorders, hyperactivity, skin rashes, poor bone strength, and an abnormally small head.

For a complete picture of Phenylketonuria (PKU)’s pipeline drug market, buy the report here.

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s pipeline drugs offers detailed profiles of pharmaceutical drugs in all stages of pre-clinical and clinical development, from discovery through to pre-registration. Coverage is limited to novel human medicinal drugs and biosimilars seeking market approval proprietary and is one of two primary repositories of pharmaceutical drug information offered by GlobalData through its Pharmaceutical Intelligence Center.