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.
cystetic Medicines doses first healthy volunteer in CM001 Phase one clinical trial
First Patient Enrolled in Phase 3 Clinical Trial Evaluating ARINA-1 in Lung Transplant
Insmed completes adult patient enrolment in brensocatib pivotal ASPEN study
Aluna Raises USD 15.3m in Series B Funding
CF Foundation Invests Up to USD 15m in ReCode Therapeutics for Development of mRNA Therapy
Markels Named to Arcturus Therapeutics Board of Directors
SpliSense commences first-in-human, Phase 1/2 clinical trial of SPL84 to treat cystic fibrosis
Arcturus Forges Collaboration with CSL to Develop and Commercialize Self-amplifying mRNA Vaccines
Vanda Pharmaceuticals VPO-227 granted US FDA Orphan Drug Designation for the treatment of cholera