Update for July 7, 2020

Regulatory Update
 
The FDA approved triheptanoin (Dojolvi, Ultragenyx) on 6/30/2020 as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). Ultragenyx set WAC for triheptanoin at $4,875 per 500 ml vial, which indicates an average annual cost of $138,000.
 
The FDA has approved remimazolam (Byfavo, Acacia) on 7/2/2020 for the induction and maintenance of procedural sedation in adults undergoing surgical procedures lasting 30 minutes or less.
 
The FDA approved fostemsavir (Rukobia, ViiV Healthcare), on 7/3/2020, in combination with other antiretrovirals, for the of treatment heavily treated adults with multidrug-resistant HIV-1 infection failing their current antiretroviral regimen due to resistance, intolerance, or safety considerations.
 
The FDA rejected Heron Therapeutics Bupivacaine, Meloxicam (HTX-011) for a second time and requested additional data regarding exposure of excipients in preclinical reproductive toxicology studies and a process change for the allowable level of an impurity during manufacturing.
 
Mezzion submitted an NDA for udenafil to improve the physiology of patients 12 years of age and older with single ventricle heart disease (SVHD) who have undergone a Fontan operation.
 
The FDA designated Nodic Nanovector Lutetium (177lu) lilotomab satetraxetan, a Fast Track therapy for treatment of adults with relapsed or refractory marginal zone lymphoma who received at least two prior systemic therapies.
 
Announced Research Updates
 
Iterum Therapeutics announced that in the 1,670 patient, Phase III SURE 1 trial, oral sulopenem/probenecid for five days did not meet non-inferiority for test of cure compared to oral ciprofloxacin for three days (66.8% vs 78.6%) in the treatment of uncomplicated urinary tract infections in patients infected with quinolone-susceptible bacteria. However, in patients infected with quinolone-resistant organisms, sulopenem/probenecid was superior to ciprofloxacin at the test of cure visit (62.6% vs 36%).
 
Hua announced that in a 24-week, 766 patient, Phase III trial, dorzagliatin added to metformin lowered HbA1c 0.66% compared to metformin monotherapy (a decrease of 1.02% vs 0.36%) in Chinese patients with type 2 diabetes.
 
Helsinn and MEI Pharma discontinued a Phase III trial evaluating pracinostat in combination with azacitidine in the treatment of acute myeloid leukemia patients who are unfit to receive standard intensive chemotherapy, when a futility analysis revealed little chance of treatment success.
 
ObsEva announced that in the 24-week, 526 patient, Phase III PRIMROSE 2 trial, heavy menstrual bleeding was reduced to 80/ml or less with the reduction being at least 50% from baseline in 75.5% of patients treated with linzagolix 200 mg and hormone add-back therapy and 56.4% with linzagolix 100 mg monotherapy compared to placebo in patients with uterine fibroids and heavy menstrual bleeding.
 
Published Research Updates
 
An analysis of post-marketing data from Japan found that 12.6% of 6,451 patients that took tofogliflozin experienced an ADR, with the most commonly reported events being hypoglycemia, polyuria/pollakiuria, volume depletion-related disorders, urinary tract infections, genital infections and skin diseases.
 
In a 52-week, 62 patient, Phase III trial, treatment with weekly somapacitan was similar to daily growth hormone in the reduction of visceral, subcutaneous and total adipose tissue in Japanese adults with growth hormone deficiency