Two Approvals, One new QIDP, One rejection and a BLA withdrawal

FDA Actions

The FDA approved solriamfetol (Sunosi, Jazz Pharmaceuticals) on 3/20/2019 to improve wakefulness in adults with excessive daytime sleepiness associated with narcolepsy or obstructive sleep apnea.

The FDA approved brexanolone (Zulresso, Sage Therapeutics)on 3/19/2019 for the treatment of postpartum depression. 

The FDA granted Qualified Infectious Disease Product (QIDP)to the oral and IV formulations of sulopenem in March 2019.  

The FDA rejected sotagliflozin on 3/22/2019.Lexicon and Sanofi are working with the FDA to determine future development of the drug. 

Spectrum withdrew the BLA for eflapegrastimto provide more information to the FDA on the chemistry, manufacturing and controls process.

Two Trials Published

In a draft cost-effectiveness analysis, ICER estimated a quality-adjusted life year of $826,000 for use of siponimod in the treatment of secondary progressive multiple sclerosis, assuming the same cost for siponimod as ocrelizumab.

In a 48-hour, 389 patient, Phase III trial, 50% of Trevena’s oliceridine 0.1 mg patients, 62% of 0.35 mg patients and 65.8% of 0.5 mg patients reached the combination endpoint of a 30% or > improvement in pain (SPID-48), received no rescue drugs, completed the study period and did not reach dosing limits compared to with 15.2% that received placebo in patients with moderate-to-severe pain following bunionectomy. The 0.35 mg and 0.5 mg doses were non-inferior to morphine in reaching the primary endpoint. In a 389 patient, Phase III trial, GI ADR increased in a dose-dependent manner in oliceridine regimens; 40.8% with 0.1 mg, 59.5% with 0.35 mg and 70.9% with 0.5 mg compared to 24.1% with placebo and 72.4% with morphine.

Five Announced Investigational Drug Developments

1. Demira announced that in a 16-week, 280 patient, Phase Ib trial, lebrikizumab improved the EASI score by 62.3% in patients receiving 125 mg every four weeks, 69.2% with 250 mg every four weeks and 72.1% for patients receiving 250 mg every two weeks compared to a 41.1% improvement with placebo in patients with moderate-to-severe atopic dermatitis. Dermira plans to initiate Phase III trials at the end of 2019.
2. Urovant announced that in the 12-week, 1,518 patient, Phase III, EMPOWUR trial, treatment with vibegron reduced micturitions per 24 hours and urge urinary incontinence episodes per 24 hours compared to placebo in patients with overactive bladder. Vibegron was no different than tolterodine in the trial. Compared to placebo, vibegron reduced both primary endpoints as early as two weeks. Urovant enrolled 507 patients from the EMPOWUR trial in a 40-week RCT extension trial to evaluate the safety of longer-term treatment of vibegron. The FDA accepted the BLA for AR101 and set a PDUFA date for late January 2020.
3. GenNeuro announced that in the ANGEL-MS trial, a 96-week, 154 patient, extension of the Phase IIb CHANGE-MS trial, where the highest dose of temelimab (18mg/kg) had 2.4% of patients develop worsening of 20% or more in the Timed 25-Foot Walk Test compared to 10.2% with placebo in patients with multiple sclerosis. There was no statistical difference in worsening in neurological disability between temelimab and placebo.
4. Biogen and Eisai announcedthat an interim analysis by an independent committee of the Phase III aducanumab trials ENGAGE and EMERGE involving patients with mild cognitive impairment due to Alzheimer's disease and mild Alzheimer's disease dementia were unlikely to meet their primary endpoints resulting in a discontinuation of both studies. Biogen also discontinued a Phase II safety trial and long-term extension of a Phase Ib trial. Biogen is considering whether to initiate a Phase III secondary prevention trial.
5. Conatus announced that in the 72-week, 318 patient, Phase IIb, ENCORE-NF trial, emricasan did not improve the CRN fibrosis stage without suffering a worsening of steatohepatitis compared to placebo in patients with biopsy-confirmed nonalcoholic steatohepatitis (NASH) and liver fibrosis.