Proteostasis Gains After Cystic Fibrosis Drug Is Granted Orphan Status

Micro-cap biotech Proteostasis Therapeutics Inc PTI 4.67% was up strongly Tuesday morning.

What Happened

Proteostasis, which focuses on therapies for dysfunctional protein processing, specifically for cystic fibrosis, announced Tuesday its PTI-428 has been granted orphan drug designation, or ODD, by the European Commission, for treating cystic fibrosis.

PTI-428 is the Boston-based biotech’s lead product candidate. It’s the company’s proprietary cystic fibrosis transmembrane conductance regulatory amplifier in clinical development.

The pipeline asset has also secured ODD, Breakthrough Therapy Designation and Fast Track Designation from the U.S. Food And Drug Administration.

Why It’s Important

Orphan drug designation is granted by the EU based on a positive opinion by the EMA’s Committee’s Orphan Medical Products, bestowing regulatory and financial incentives for companies developing therapies to treat serious disorders affecting no more than five in 10,000 persons in the EU.

The notable among the benefits are 10-year market exclusivity in the EU upon approval, eligibility for protocol assistance, reduced fees and access to the EU’s centralized marketing authorization procedure.

What Next

Proteostasis said it will advance PTI-428 in the clinic later this year, as part of the planned 28-day Phase 2 studies of its proprietary combination CFTR modulator treatments.

https://www.benzinga.com/general/biotech/19/06/13857111/proteostasis-therapeutics-rallies-after-cystic-fibrosis-drug-is-granted-orphan-designation