An FDA review of Mallinckrodt’s terlipressin questioned whether clinical data on hepatorenal syndrome type 1 reversal, as demonstrated by a decrease in serum creatinine, demonstrated decreased mortality, need for dialysis or intensive care days. However, the FDA Cardiovascular and Renal Drugs Advisory Committee voted 8-7 to recommend approval in July 2020. The FDA was not convinced and rejected the NDA for terlipressin and requested data to support a positive risk-benefit profile for terlipressin for HRS-1 patients in September 2020.
The FDA granted BeyondSpring’s plinabulin a Breakthrough Therapy Designation for the prevention of chemotherapy-induced neutropenia.
The FDA requested that Sarepta use a different potency assay when manufacturing its microdystrophin gene therapy, before using the drug in a Phase III trial.
The EMA requested 52-week data for 134 patients enrolled in a Phase III trial of BioMarin’s valoctocogene roxaparvovec for the treatment of Hemophilia A.
Announced Research Updates
Merck announced that gefapixant 45 mg BID reduced the 24-hour coughs per hour average 18.45% more than placebo in the 12-week, 730 patient, Phase III, COUGH-1 trial and by 14.64% more than placebo in the 24-week, 1,314 patient, Phase III, COUGH-2 trial in patients with refractory or unexplained chronic cough. Treatment with gefapixant 15 mg BID did not differ from placebo in neither the COUGH-1 nor COUGH-2 trials. Taste-related adverse effects were frequent with the 45 mg dose with an incidence of 58% in COUGH-1 and 68.6% in COUGH-2.
Corbus announced that in the 52-week, 365 patient, Phase III, RESOLVE-1 trial, treatment with lenabasum did not improve the American College of Rheumatology Combined Response Index for Systemic Sclerosis (ACR CRISS) scores compared to placebo in patients with diffuse cutaneous systemic sclerosis.
Gilead and Galapagos announced that in the 156-week, 739 patient, Phase II, DARWIN 3 trial (extension trial for Darwin I and II), there was an increase in infections with filgotinib, but no other safety issues.
Published Research Updates
In a 52-week, 121 patient, Phase III trial, treatment with vosoritide increased annualized growth velocity by 1.57 cm/year more than placebo in children (ages 5 to 18) with achondroplasia.
In the 24-week, 256 patient, Phase II, WILLOW trial, treatment with brensocatib resulted in the time to the first exacerbation of 134 days with 10 mg and 96 days with 25 mg compared to 67 days with placebo in patients with non-cystic fibrosis bronchiectasis
In the 266 patient, Phase III FRESCO trial, the most common adverse events with fruquintinib were hypertension, palmar-plantar erythrodysesthesia syndrome and proteinuria.
In the 679 patient, Phase III TARGET trial, 91.3% of patients treated with ceftobiprole achieved a 20% or more reduction from baseline in lesion size at 48 to 72 hours compared to 88.1% of patients treated with vancomycin plus aztreonam in patients with acute bacterial skin and skin structure infection.
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