The FDA rejected teplizumab to delay the development of type 1 diabetes in at-risk individuals because, as stated in the FDA review, the PK/PD bridging study was not adequate to demonstrate pharmacokinetic equivalence between the AGC Biologics manufactured templizumab and the Lilly manufactured drug used in early studies. New PK/PD data will be provided from the patient population in the ongoing Phase 3 PROTECT trial by the end of 3Q21. Manufacturing problems were also noted in the rejection, but Prevention Bio feels these have been rectified in data recently provided to the FDA.
ChemoCentryx provided additional information for avacopan to the FDA to answer questions from the FDA and its Arthritis Advisory Committee. The information is considered a major addendum to the NDA, so the PDUFA date has been moved back three months to 10/7/2021.
The FDA notified Iterum the NDA for sulopenem etzadroxil/probenecid contained unspecified deficiencies that would prevent discussions with the company on labeling and post marketing requirements. Iterum did not know if this would change the July PDUFA date for sulopenem etzadroxil/probenecid.
Gilead submitted an NDA for lenacapavir for the treatment of HIV-1 infection in heavily treatment-experienced patients with multi-drug resistant HIV-1 infection.
Announced Research Updates
GenSight announced that in the 78-week, 98 patient, Phase III, REFLECT trial, patients that received an injection of lenadogen nolparvovec in both eyes had a greater visual acuity improvement (12 ETDRS letter improvement and 8 letter improvement) compared to patients that received the active drug and sham injection (8 ETDRS letter improvement and 4 ETDRS letter improvement) in patients with LHON caused by a mutated ND4 mitochondrial gene.
Published Research Updates
In a 4-week, 20-patient, open-label, Phase II trial, treatment with odevixibat reduced serum bile acids, and improved pruritus and sleep disturbance in children with cholestatic diseases. 13 of the 20 patients had a diagnosis of familial intrahepatic cholestasis.
In the 40-week, 478 patient, Phase III, SURPASS-1 trial, treatment with tirzepatide lowered HbA1c 1.87% with 5 mg, 1.89% with 10 mg and 2.07% with 15 mg compared to a 0.04% increase with placebo in patients with type 2 diabetes.
In the 24-week, 269 patient Phase IIb HEADWAY trial, intepirdine did not improve the Unified Parkinson's Disease Rating Scale-Part III score compared to placebo patients with dementia with Lewy bodies.
In a 15-day, 150 patient, Phase III trial, patients treated with zuranolone had a 17.8 point reduction in their HAMD-17 total score compared to a 13.6 point decrease with placebo in patients with postpartum depression.
In a 13.9-month, 54 patient, Phase II trial, adding TLPLDC to standard of care resulted in overall survival (OS) of 76.5% and progression-free survival (PFS) of 57.1% in 28 patients in a crossover cohort of patients with recurred melanoma from a Phase IIb TLPLDC trial and OS of 85.7% and PFS of 52.2% in a cohort of 26 patients with metastatic melanoma.
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