New Drug Update for The Week Ending May 11

Regulatory Updates

The FDA accepted the NDA for bempedoic acid and a combination bempedoic/ezetimibe tablet in May 2019 and set a PDUFA date of 2/21/2020 for bempedoic acid and 2/26/2020 for the combination.

The FDA granted cannabidiol gel Fast Track Status for the treatment of Fragile X syndrome in May 2019.

The FDA granted leronlimab Fast Track status in May 2019 for use in combination with carboplatin for the treatment of patients with CCR5-positive metastatic triple-negative breast cancer.

The EMA approved volanesorsen as adjunct treatment of familial chyromicronemia syndrome (FCS) after diet adjustment and triglyceride therapy in May 2019.

Announced Research Updates

Voyager announced that in an open label, 1-year, 8 patient, Phase I trial, treatment with VY-AADC increased on-time without dyskinesia by 1.7 hours, decreased off-time by 2.2 hours and decreased the Parkinson's Disease Questionnaire (PDQ-39) score by 7.6 points in patients with Parkinson's disease.

Alder announced that in the 24-week, 1,072 patient, Phase III, PROMISE-2 trial, eptinezumab reduced monthly migraine days 2.1 to 2.6 days more than placebo and 57% reported much/very much improvements in their most bothersome symptom compared to 41% with placebo.

Published Research Update

In a 3.2-year, 105 patient, Phase I/II, open label, dose ranging trial, treatment with guadecitabine resulted in an overall response (a composite of complete response, partial response, marrow complete response, and hematological improvement) in 40-51% of patients with hypomethylating agent treatment-naive or relapsed or refractory patients with intermediate-risk or high-risk myelodysplastic syndromes.

In a 3-month, 46 patient, Phase I/II, dose ranging trial, RG6042 reduced mutant huntingtin protein levels in the CSF by 20-38% compared to placebo in patients with early stage Huntington's disease.

In the 27.4-month, 1,015 patient, phase III, TRINOVA-3/ENGOT-ov2/GOG-3001 trial, patients received six cycles of trebanaib/paclitaxel/carboplatin or placebo, followed by trebanaib or placebo maintenance therapy for 18 months. Median progression free survival (PFS) did not differ between trebanaib and placebo (15.9 vs 15 months).

In a 48-week, 48 patient, Phase II trial, 99% of patients treated with ublituximab achieved at least a 95% reduction in peripheral CD19+ B cells within 2 weeks after the second infusion on day 15 in patients with relapsing forms of multiple sclerosis.

In a 106 hepatorenal syndrome (HRS) patient trial, 48% of patients treated with terlipressin and albumin had a reversal of HRS type 1 (rapidly progressive loss of renal function) and 46% had a reversal of HRS type 2 (chronic ascites and more moderately elevated renal parameters).