Weekly Update for the Week Ending November 23, 2019

Regulatory Update
 
The FDA approved givosiran (Givlaari, Alnylam) on 11/20/2019 for the treatment of adults with acute hepatic porphyria. WAC for givosiran is $39,000 per vial with an estimated annual cost after discount of $442,000.
 
The FDA approved cenobamate (Xcopri, SK Life Science) on 11/21/2019 for the treatment of partial-onset seizures in adult patients with epilepsy. The FDA reports the most common ADR in the trials were somnolence, dizziness, fatigue, and diplopia. Cenobamate may cause a reduction in the QT interval > 20 milliseconds, so the FDA advises avoiding the drug in patients with Familial Short QT syndrome.
 
The FDA has designated arimoclomol a Breakthrough Therapy for Niemann-Pick Disease Type C.
 
Alkermes submitted an NDA for olanzapine/samidorphan (ALKS 3831) for the treatment of schizophrenia and for the treatment of bipolar I disorder.
 
The FDA has granted sarizotan a Rare Pediatric Disease designation for the treatment of Rett syndrome.
 
BioMarin submitted an MAA for valoctocogene roxaparvovec to treat Hemophilia A. 
 
Takeda plans to file an NDA for its Dengue vaccine by the end of 2020.
 
Announced Research Updates
 
Merck and Bayer announced that in a 5,050 patient, Phase III VICTORIA trial, vericiguat added to current heart failure therapies increased the time to the occurrence of cardiovascular death or heart failure hospitalization compared to placebo in patients with worsening chronic heart failure with reduced ejection fraction (HFrEF) following a decompensation event, defined as heart failure with hospitalization or receiving an intravenous diuretic for heart failure without hospitalization.
 
UCB announced that in the 16-week, 435 patient, Phase III, BE READY trial, more patients treated with bimekizumab achieved PASI 90 and IGA 0/1 than placebo in patients with moderate-to-severe chronic plaque psoriasis.
 
Myovant announced that in the 48-week, 934 patient, Phase III, HERO trial, 96.7% of patients treated with relugolix achieved sustained testosterone suppression to castrate levels (< 50 ng/dL) compared to 88.8% with leuprolide in men with advanced prostate cancer.
 
The Medicines Company announced that in the 18-month, 482 patient, Phase III, ORION-9 trial, the placebo adjusted reduction in LDL with inclisiran was 50% in patients with heterozygous familial hypercholesterolemia with elevated LDL despite maximally tolerated statins.
 
Published Research Updates
 
In the 26-week, 400 patient, Phase III, FUEL trial, treatment with udenafil did not increase oxygen consumption at peak exercise compared to placebo in adolescents who had undergone Fontan surgery.
 
In the 1,355 migraine patient, Phase III, ACHIEVE II trial, the percentage of patients pain free at two hours was 20.7% of those treated with ubrogepant 25 mg, 21.8% with 50 mg and 14.3% with placebo. The most bothersome symptoms at two hours were eliminated in 38.9% treated with ubrogepant 50 mg compared to 27.4% with placebo. The lower 25 mg dose of ubrogepant did not reach a statistical difference compared to placebo for freedom of the most bothersome symptoms.
 
In a 142 patient, Phase II trial, treatment with buparlisib provided limited efficacy in patients with resistant solid or hematologic malignancies with PI3K pathway activation.