WARREN, N.J. — PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the sepiapterin MAA for PKU has been validated and accepted for review by the EMA.
“The validation of the MAA is an important step towards making sepiapterin available to children and adults affected by PKU in Europe,” said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. “The European submission is the first of several global submissions planned in 2024.”
PTC expects to submit the sepiapterin NDA to the FDA no later than the third quarter of 2024. In addition, submissions are planned in a number of additional countries in 2024 including Brazil and Japan.
The sepiapterin MAA includes data from the phase 3 APHENITY trial which demonstrated 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 of <360 µmol/L, and 22% of subjects had normalization of Phe levels. The application also includes data from the APHENITY open-label extension study which provides evidence of sepiapterin durability of effect as well as data from the Phe tolerance sub-study. Phe tolerance sub-study results collected to date demonstrate that subjects are liberalizing their diet, beyond the recommended daily allowance of protein, and maintaining control of Phe within guideline target levels of <360 μmol/L. The ability to enable patients to liberalize their highly restrictive diets and still maintain Phe control is a very meaningful sepiapterin treatment benefit for patients as well as for physicians and payers.
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 Therapeutics, Inc.
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. PTC’s ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC’s mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC’s strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders.
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