Update for the week ending August 10, 2019

Regulatory Update

The FDA accepted the NDA for givosiran for the treatment of acute hepatic porphyria and set a PDFUA date of 2/4/2020.

The FDA accepted the NDA for avapritinib for the treatment of PDGFRA Exon 18 mutant gastrointestinal stromal tumors and set a PDFUA date of 2/14/2020.

After canceling an advisory committee meeting to review lumateperone due to new information provided for the NDA, the FDA delayed the PDUFA date for 3 months to 12/27/2019.

The FDAdesignated teplizumaba Breakthrough Therapy Designation for the prevention or delay of clinical type 1 diabetes in patient at risk of developing the disease.

GSK licensed its Ebola vaccines to the Sabin Vaccine Institute. Sabin has signed an agreement with the National Institute of Allergy and Infectious Diseases’s (NIAID) Vaccine Research Center to develop the drug.

The EMA has accepted the MAA for abiciparfor the treatment of neovascular age-related macular degeneration.

DBV withdrew itsoriginal BLA for the Viaskin peanut allergy transdermal patch in 2018 due to FDA concerns about its manufacturing process. DBV resubmitted a BLA in August 2019.

Announced Research Updates

Rhythm announced that in a 12-month, 11 patient, Phase III, open-label trial, treatment with setmelanotide resulted in a 10% reduction in body weight with a mean body weight reduction of 25.4% in eight patients with pro-opiomelanocortin deficiency obesity. Rhythm announced that in a 12-month, 10 patient, Phase III, open-label trial, treatment with setmelanotide resulted in a 10% reduction in body weight with a mean body weight reduction of 25.4 in five patients with leptin receptor deficiency obesity. Patients in both trials went through a withdrawal period, where weight was gained back before starting back on the drug for an extension trial. Patients lost weight after restarting the drug. Rhythm Pharmaceuticals plans to complete a rolling NDA submission for setmelanotide for the treatment of both pro-opiomelanocortin and leptin receptor deficiency obesity in 4Q19. Rhythm has ongoing extension trials for both pro-opiomelanocortin and leptin receptor deficiency obesities. Rhythm also continues to enroll portents for both deficiencies and is targeting pediatric patients.  

Basileaannounced that 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.
 
Published Research Updates

In a 92 patient, Phase II trial, treatment with cediranib improved progression-free survival compared to placebo (7.2 months v 5.6 months) in patients with unresectable malignant pleural mesothelioma.

In the 66 patient, 3-month, Phase III APPROACH trial, volanesorsen decreased triglycerides 77% vs an 18% increase with placebo in patients with familial chylomicronemia syndrome.

Arena announced that in a 22-week, 61 patient, Phase II trial, ralinepag decreased pulmonary vascular resistance by 163.9 dyn.sec/cm5 compared to an increase of 0.7 dyn.sec/cm5 with placebo in patients with pulmonary arterial hypertension (PAH).

In a 4.9 month, 334 patient, Phase II trial, 50% of patients treated with taselisib added to letrozole achieved an objective response compared to 39% with letrozole monotherapy in postmenopausal women with ER+, HER2-operable breast cancer. However, there was no difference in pathological complete response between groups.