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01 Apr 2024
Admin
News Article

PTC Seeks European Marketing Approval for Sepiapterin

PTC Therapeutics seeks European marketing approval for sepiapterin for Treatment of phenylketonuria

Overview

PTC Therapeutics, Inc. announced the submission of the sepiapterin MAA to the European Medicines Agency. The MAA submission is for the treatment of paediatric and adult patients with phenylketonuria (PKU), including the full spectrum of disease subtypes.

Words from CEO: PTC Therapeutics

"This MAA submission is the first in a series of global submissions for sepiapterin as we move closer to bringing this therapy to children and adults with PKU worldwide," said Matthew B. Klein, M.D., chief executive officer of PTC Therapeutics. "The strength of the sepiapterin data generated in clinical trials substantiate how differentiated sepiapterin is from currently available therapies and support how sepiapterin could address the significant unmet medical need for PKU patients."

Phase 3 APHENITY Trial

The sepiapterin MAA includes the results of the phase 3 APHENITY trial in which sepiapterin had a statistically significant and clinically meaningful reduction in blood phenylalanine levels in paediatric and adult PKU patients.
Sepiapterin treatment resulted in a mean reduction in Phe levels of 63% in the overall treated population and 69% in the subgroup of subjects with classical PKU.
The vast majority of subjects (84%) achieved Phe control in accordance with treatment guidelines, and 22% of subjects had normalization of Phe levels.
Data from the ongoing APHENITY open-label extension study demonstrates that these sepiapterin treatment effects are durable.
In addition, data from the Phe tolerance sub study demonstrate that subjects are liberalizing their diet, beyond the recommended daily allowance of protein, and maintaining control of Phe within target levels from guidelines.
The ability to enable patients to liberalize their highly restrictive diets and still maintain Phe control, is a very meaningful sepiapterin treatment benefit for physicians, payers and patients.

Submission for NDA

PTC expects to submit the sepiapterin NDA to the FDA no later than the third quarter of 2024.
Submissions in 2024 are planned in a number of additional key countries where PTC has existing rare disease commercial infrastructure including Brazil and Japan.

About Sepiapterin

Sepiapterin (formerly PTC923) is an oral formulation of synthetic sepiapterin, a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in the metabolism and synthesis of numerous metabolic products.
Sepiapterin is a more bioavailable precursor than exogenously administered synthetic BH4 and has the potential to treat the broad range of PKU patients.

About Phenylketonuria

Phenylketonuria (PKU) is a rare, inherited metabolic disease, which affects the brain.
It is caused by a defect in the gene that helps create the enzyme needed to break down phenylalanine.
If left untreated or poorly managed, phenylalanine – an essential amino acid found in all proteins and most foods – can build up to harmful levels in the body.
This causes severe and irreversible disabilities, such as permanent intellectual disability, seizures, delayed development, memory loss, and behavioral and emotional problems.
Newborns with phenylketonuria initially don't have any symptoms, but symptoms are usually progressive, and damage caused by toxic levels of phenylalanine in the first few years of life is irreversible.
Diagnosis of phenylketonuria usually takes place during newborn screening programs. There are an estimated 58,000 people with phenylketonuria globally.

About PTC

PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders.