Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Study Phase : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
US FDA approves PTC Therapeutics' metabolic disorder drug
Details : Sephience (sepiapterin) is a PAH activator indicated for the treatment of hyperphenylalaninemia in adult and pediatric patients 1 month of age and older with PKU.
Product Name : Sephience
Product Type : Miscellaneous
Upfront Cash : Inapplicable
July 28, 2025
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Highest Development Status : Approved FDF
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
PTC gets Positive CHMP Opinion for Sephience in Treating PKU in Children and Adults
Details : Sephience (sepiapterin) is an oral formulation of sepiapterin, has a dual mechanism of action to increase activity of the phenylalanine hydroxylase enzyme. It is being evaluated for Phenylketonuria.
Product Name : Sephience
Product Type : Miscellaneous
Upfront Cash : Inapplicable
April 25, 2025
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
PTC Therapeutics Presents New Sepiapterin Data from Ongoing Studies
Details : PTC923 (sepiapterin) is an oral formulation, that has a dual moa to increase the activity of the PAH enzyme. It is being evaluated for the treatment of pediatric and adult phenylketonuria patients.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
March 20, 2025
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
PTC’s Sepiapterin NDA Accepted by FDA for Phenylketonuria
Details : PTC923 (sepiapterin) is an oral formulation, that has a dual moa to increase the activity of the PAH enzyme. It is being evaluated for the treatment of pediatric and adult phenylketonuria patients.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
October 01, 2024
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
PTC Announces Submission of Sepiapterin MAA for Treatment of PKU to EMA
Details : PTC923 (sepiapterin) is a precursor to intracellular tetrahydrobiopterin that acts as nitric oxide synthase modulator. It is being evaluated for the treatment of Phenylketonuria.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
March 28, 2024
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Details : Sepiapterin is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Phenylketonurias.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
March 08, 2024
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
PTC Therapeutics Announces APHENITY Trial Achieved Primary Endpoint with Sepiapterin in PKU Patients
Details : PTC923 (sepiapterin) is a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in the metabolism and synthesis of numerous metabolic products and has the potential to treat the broad range of PKU patients.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
May 17, 2023
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
Details : PTC923 is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Phenylketonurias.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
December 21, 2021
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Study of PTC923 in Participants With Phenylketonuria
Details : PTC923 is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Phenylketonurias.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
October 29, 2021
Lead Product(s) : Sepiapterin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Sepiapterin
Therapeutic Area : Endocrinology
Study Phase : Phase II
Sponsor : PTC Therapeutics
Deal Size : $366.0 million
Deal Type : Acquisition
PTC Therapeutics to Acquire Censa Pharmaceuticals
Details : Censa Pharmaceuticals acquisition diversifies and strengthens PTC 's portfolio of rare disorders by adding the lead orphan metabolic disease drug, CNSA-001.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : $10.0 million
May 06, 2020
Lead Product(s) : Sepiapterin
Therapeutic Area : Endocrinology
Highest Development Status : Phase II
Sponsor : PTC Therapeutics
Deal Size : $366.0 million
Deal Type : Acquisition
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