Proteostasis Therapeutics Inc (NASDAQ: PTI) is grabbing the attention of investors with premarket gains of more than 20%. The gains come after the company made an announcement with regard to the receipt of orphan drug designation.
PTI Receives Orphan Drug Designation In Europe
The news broke via press release early this morning. In the release, Proteostasis Therapeutics said that the European Commission, also known as the EC, has granted orphan drug designation to PTI-428.
PTI-428 is being developed as a treatment for cystic fibrosis. The treatment is known as a cystic fibrosis transmembrane conductance regulator, or CFTR amplifier. Currently, PTI-428 is in the midst of clinical devlopment.
However, the orphan drug designation from the EC isn’t the only positive feedback that PTI has received from a regulatory body surrounding the drug. Let’s not forget, the United States Food and Drug Administration has granted Breakthrough Therapy designation and Fast Track designation to the treatment as well.
The orphan drug designation is the result of a positive opinion from the European Medicines Agency’s Committee for Orphan Medical Products. As a result of the designation, should the drug be approved, the company will enjoy 10-year market exclusivity in the EU, reduced fees associated with regaulatory filings, and more.
Why This Is So Important
Aside from the fact that orphan drug designation ultimately shortens the path toward potential regulatory approval and provides 10-year market exclusivity, it validates the work that PTI is doing to develop the treatment. All in all, this is very exciting news for investors.
This designation by the EC is further validation of both PTI-428’s potential and PTI’s mission to offer additional disease modifying treatment options for CF. Orphan drug status is granted to development-stage drugs that make a major contribution to patients’ care, either by demonstrating a significant clinical benefit over existing therapies or by providing a treatment for patients for whom existing therapies do not work. We look forward to advancing PTI-428 in the clinic later this year, as part of the planned 28-day Phase 2 studies of our proprietary combination CFTR modulator treatments. – Meenu Chhabra, President and Chief Executive Officer of Proteostasis Therapeutics.
What We’ll Be Watching For Ahead
The CNA Finance team will continue to follow PTI incredibly closely. In particular, we’re interested in following the continued development of PTI-428 as if approved in the United States and Europe, the treatment could lead to substantial revenue for the company. Nonetheless, we’ll continue to follow the story closely and bring the news to you as it breaks!
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