The FDA approved bempedoic acid (Nexletol, Esperion) on 2/21/20 as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease (ASCVD). Esperion plans to price bempedoic acid around $10 a day. Bempedoic acid will be commercially available on 3/30/2020 and Esperion will offer a prescription reduction program that will reduce the copay to $10 for up to a 3-month supply.
The FDA approved eptinezumab (Vyepti, Lundbeck) on 2/21/20 for migraine prevention in adults. Eptinezumab is administered as a 30-minute IV infusion every 3 months. Lundbeck plans to have eptinezumab available in April 2020.
The FDA approved meloxicam injection (Anjeso, Baudax Bio) for the treatment of moderate to severe pain in adults, as monotherapy or concomitantly with other non-NSAID analgesics. Meloxicam injection is given as a once-daily intravenous injection.
The FDA accepted the BLA for valoctocogene roxaparvovec and set a PDUFA date of 8/21/2020. BioMarin indicated it may change between $2 million and $3 million for a dose of valoctocogene roxaparvovec.
The FDA accepted the NDA for lurbinectedin for the treatment of small cell lung cancer that relapsed after platinum-based therapy and set a PDUFA date of 8/16/2020.
The FDA accepted the NDA for berotralstat for the prevention of hereditary angioedema (HAE) attacks and set a PDUFA date of 12/3/2020.
An FDA advisory committee will review Viaskin Peanut patch on May 15.
Bluebird delayed its launch of beta beglogene darolentivec in Europe until February 2020 in order to finalize large scale manufacturing. The FDA requested additional information on beta beglogene darolentivec; Bluebird has delayed submission of a BLA to the end of 2020.
Announced Research Updates
BrainStorm is evaluating mesenchymal stem cells neurotrophic growth factors (MSC-NTF) in a 200 patient, Phase III trial as a treatment for faster-progressing amyotrrophic lateral sclerosis (ALS) and an open-label Phase II, progressive multiple sclerosis (MS) trial. BrainStorm expects topline results from both trials to be available in 4Q20.
NGM announced that in a 24-week, 78 patient, Phase II trial, treatment with aldafermin resulted in a 39% decrease in liver fat content compared to 13% decrease with placebo in patients with biopsy-confirmed NASH with F2-F3 liver fibrosis.
Published Research Updates
The 204 patient, Phase III, ENDEAVOR trial was discontinued after an average follow-up of 6.7 months when mortality was higher with revusiran than placebo (12.9% vs 3.0%) in patients with cardiomyopathy caused by hereditary transthyretin-mediated amyloidosis.
In a 4-week, 70 patient, Phase II trial, treatment with nemolizumab reduced the pruritus scale score by 4.5 points compared to a 1.7 point decrease with placebo in patients with moderate-to-severe prurigo nodularis and severe pruritus.
In a 78 patient Phase II trial, tivantinib plus cetuximab did not improve the overall response rate compared to cetuximab monotherapy in patients with cetuximab-naive, platinum-refractory head and neck squamous cell carcinomas.
In the 4-week, 71 patient, Phase II, PROGRESS-HF trial, treatment with elamipretide did not improve left ventricular end systolic volume compared to placebo in patients with heart failure with reduced ejection fraction (HFrEF).
In a 42-day, 227 patient, Phase IIb trial, treatment with sofpironium resulted in at least a one-grade improvement in the Hyperhidrosis Disease Severity Measure-Axillary score in 70% of patients treated with 5% gel, 79% with a 10% gel, 76% with a 15% gel compared to 54% with placebo in patients with axillary hyperhidrosis.
In the 12-week, 1,518 patient, Phase III, EMPOWUR trial, treatment with vibegron reduced micturitions per 24 hours by 1.8 episodes/day compared to a 1.3 episode decrease with placebo and a 1.6 episode decrease with tolterodine (non-significant difference) in patients with overactive bladder.
In a 24-month, 275 patient, Phase IIb trial, treatment with nelipepimut-S plus sargramostim did not improve disease-free survival compared to sargramostim monotherapy in patients with HER2 low-expressing breast cancer who were receiving trastuzumab.
In the 12-week, 888 patient, Phase III, PROMISE-1 trial, eptinezumab reduced monthly migraine days by 4.0 days with 30 mg, 3.9 days with 100 mg and 4.3 days with 300 mg compared to a 3.2 days reduction with placebo in patients with episodic migraine.
In a 113 patient, Phase I, open-label trial, treatment with mirvetuximab soravtansine and bevacizumab resulted in an ORR of 39% and median PFS of 6.9 months in patients with FRα-positive, platinum-resistant ovarian cancer.
In a 42 patient, Phase II trial, 52.4% of patients treated with lurbinectedin achieved progression free survival at 12 weeks in patients with progressive malignant pleural mesothelioma.
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