Although it was a short week due to the Labor Day holiday, the FDA maintained a steady pace of business.
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Although it was a short week due to the Labor Day holiday, the U.S. Food and Drug Administration maintained a steady pace of business.
Sept. 8
London-based Silence Therapeutics announced the FDA granted Fast Track designation to SLN124, a novel siRNA targeting the TMPRSS6 gene, for the treatment of polycythemia vera. SLN124 is designed to address several hematological diseases by targeting TMPRSS6 to increase endogenous hepcidin. SLN124 demonstrated proof of mechanism and was well tolerated in a healthy volunteer study completed last year. The company plans to start a phase I/II study in PV patients this year.
Sept. 7
Pfizer received Breakthrough Therapy designation for GBS6 (PF-06760805), the company’s investigational Group B Streptococcus (GBS) vaccine candidate. The vaccine is being developed for the prevention of invasive GBS disease due to the vaccine serotypes in newborns and young infants by active immunization of their mothers during pregnancy.
California-based AnMax Bio announced the FDA granted AMB-05X Fast Track designation for the treatment of tenosynovial giant cell tumor. A monoclonal antibody, AMB-05X, targets serious macrophage-driven inflammatory, fibrotic and neovascular diseases.
Following the issuance of a Complete Response Letter in 2019, Vanda Pharmaceuticals announced a notice of opportunity for an FDA hearing on the company’s supplemental New Drug Application (sNDA) for Hetlioz (tasimelteon) to treat the jet lag disorder. In July, Vanda submitted a request under section 505(d) of the Federal Food, Drug, and Cosmetic Act for a hearing on whether the sNDA is approvable.
Tarsus Pharmaceuticals submitted an NDA to the FDA for TP-03 (lotilaner ophthalmic solution) developed for the treatment of Demodex blepharitis, a highly prevalent eyelid margin disease with no FDA-approved treatment options. The NDA includes positive data from two studies, Saturn-1 and Saturn-2. TO-03 hit all endpoints in both studies.
Praxis Precision Medicines announced it will initiate the PRAX-222 EMBRAVE clinical study for the treatment of pediatric patients with early-seizure-onset SCN2A developmental and epileptic encephalopathy after the FDA cleared its Investigational NDA for the initial dose cohort.
Sept. 6
The FDA cleared Tenaya Therapeutics’ Investigational New Drug application (NDA) for clinical testing of TN-301, a potential treatment for heart failure. TN-301 is the company’s highly selective small molecule inhibitor of histone deacetylase 6 (HDAC6) that will be developed for the potential treatment of heart failure with preserved ejection fraction (HFpEF). Tenaya has initiated a Phase I study.
California-based Eiger BioPharmaceuticals announced that the FDA informed it that it is unable to determine if the criteria for the submission of an application and issuance of an Emergency Use Authorization has been met for peginterferon lambda, a potential treatment for mild- to moderate COVID-19 infection. The company and FDA will remain in discussions over this matter.
Moleculin Biotech’s WP1122, an investigational treatment for glioblastoma multiforme, received Orphan Drug designation.
GSK announced the FDA will convene for a meeting of the Cardiovascular and Renal Drugs advisory committee to review the company’s NDA for daprodustat, an oral hypoxia-inducible factor prolyl hydroxylase inhibitor being assessed as a potential treatment for anemia due to chronic kidney disease. The adcomm meeting is set for Oct. 26.
