Benzinga | May 26, 2021 08:06AM EDT

PTC Therapeutics Reports Orphan Drug Designations Of PTC923 For Treatment Of Hyperphenylalaninemia

PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that both the United States Food and Drug Administration (FDA) and European Commission (EC) have granted Orphan Drug Designation (ODD) for PTC923 for the treatment of patients with hyperphenylalaninemia. Phenylketonuria (PKU) accounts for 98% of all hyperphenylalaninemia cases and is a metabolic condition that can lead to cognitive disabilities and seizures. 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. PTC expects to initiate a placebo-controlled Phase 3 global registrational trial, APHENITY, for patients with PKU in mid-2021.

"We are very pleased that orphan drug designations have been granted by both the US and European regulatory bodies," said Stuart W. Peltz, Ph.D. Chief Executive Officer PTC Therapeutics. "PKU is a devastating disease that can have severe and irreversible outcomes, including seizures, intellectual disability, behavioral problems and psychiatric disorders. There still remains a high unmet need for more effective and safe treatment options for patients living with PKU."

In the US, ODD is granted by the FDA's Office of Orphan Products Development to promote the development of products that may offer therapeutic benefits for diseases with a prevalence of fewer than 200,000 individuals per year. Orphan drug designation provides opportunities for grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States, if granted FDA approval.

Similarly, the European Medicines Agency's Orphan Medicinal Product Designation is designed to promote the development of drugs that may provide significant benefit for patients suffering from rare, life-threatening diseases. Each indication with an orphan designation confers ten years of market exclusivity for the particular indication in the EU if it maintains orphan designation at the time of marketing authorization.1