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PTC Therapeutics, Inc. PTCT announced that both the FDA and the European Commission (EC) have granted an Orphan Drug designation to its pipeline candidate PTC923 which is being developed for the treatment of patients with hyperphenylalaninemia.
The company plans to begin a placebo-controlled phase III registrational study – APHENITY – in mid-2021, evaluating PTC923 for addressing patients with phenylketonuria (“PKU”), which accounts for 98% of all hyperphenylalaninemia cases.
Notably, the Orphan Drug designation is granted to drugs that are capable of treating rare diseases which affect less than 200,000 people in the United States. This tag also makes the company entitled to certain other benefits, including tax credits related to clinical trial expenses, an exemption from the FDA user fee and seven-year marketing exclusivity upon potential approval.
Also, the European Medicines Agency usually grants Orphan Medicinal Product status to drugs that target serious or life-threatening conditions in the region. Additionally, every indication will enjoy a period of 10 years of market exclusivity in the EU, if approved.
Shares of PTC Therapeutics have plunged 34.7% so far this year compared with the industry’s decrease of 11.3%.
Per the company, PKU is a metabolic condition which can lead to cognitive dysfunction, behavioral problems, seizures and psychiatric disorders. Hence, if successfully developed and upon potential approval, PTC923 can serve an area of high unmet medical need.
We remind investors that Swiss pharma giant Roche RHHBY is developing Evrysdi (risdiplam) in collaboration with the SMA Foundation and PTC Therapeutics. The drug is approved for the treatment of spinal muscular atrophy (“SMA”) in the United States as well as in Europe.
PTC Therapeutics receives milestone payments from Roche from the sale of Evrysdi in the United States.
SMA is a severe, progressive neuromuscular disease that causes muscle weakness and progressive loss of movement, affecting one in 10,000 babies, and is the leading genetic cause of infant mortality.
However, competition is stiff in the SMA market from the likes of Biogen’s BIIB Spinraza and Novartis’ NVS gene therapy, Zolgensma.
PTC Therapeutics currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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