Therapy Areas: Hereditary Disorders
USFDA grants orphan drug designation to Proteostasis Therapeutics for PTI-428
16 March 2018 -

US Food and Drug Administration (USFDA) has granted orphan drug designation to United States-based Proteostasis Therapeutics for its PTI-428, a cystic fibrosis transmembrane conductance regulator amplifier drug candidate, it was announced yesterday.

The FDA Office of Orphan Products Development offers designation to products for the treatment of rare diseases or conditions affecting fewer than 200,000 patients in the US. The designation enables the sponsor of the drug to be eligible for various incentives, including a seven-year period of United States marketing exclusivity upon regulatory approval of the drug and tax credits for clinical research costs, annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

PTI-428 is an investigational CFTR amplifier in development for the treatment of CF in patients who are homozygous for the F508del mutation in the CFTR gene as an add-on therapy to approved CFTR modulators, or in conjunction with PTI-808, a potentiator, and PTI-801, a corrector.

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