PTC Therapeutics Announces Orphan Drug Designations of PTC923 for the Treatment of Hyperphenylalaninemia
SOUTH PLAINFIELD, N.J., May 26, 2021 /PRNewswire/ -- 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
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PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; the enrollment, conduct and results of PTC's PTC923 clinical trial for PKU; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; and the factors discussed in the "Risk Factors" section of PTC's most recent Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.
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1 European Medicines Agency. (2020, May 13). Market exclusivity: Orphan medicines. Retrieved April 19, 2021, from https://www.ema.europa.eu/en/human-regulatory/post-authorisation/orphan-medicines/market-exclusivity-orphan-medicines
SOURCE PTC Therapeutics, Inc.
Company Codes: NASDAQ-NMS:PTCT