August Drug Approvals and Last Week's Clinical Update

Looking back at August, we saw seven approvals and three rejections. One drug was recommended for approval and assigned a PDUFA date. Three other drugs were granted Priority Review with future PDUFA Dates. 

• The FDA approved mogamulizumab (Poteligeo from Kyowa Kirin) on 8/8/18 to treat adult patients with relapsed or refractory mycosis fungoides or Sézary syndrome after at least one prior systemic therapy. The drug has a Breakthrough Therapy Priority Designation.
• The FDA approved Amicus Therapeutics’ Galafold (migalastat), the first oral medication for the treatment of adults with Fabry disease. It’s indicated for adults with Fabry disease who have a genetic mutation determined to be responsive (“amenable”) to treatment with Galafold based on laboratory data. Fabry disease is a rare and serious genetic disease that results from buildup of a type of fat called globotriaosylceramide (GL-3) in blood vessels, the kidneys, the heart, the nerves and other organs. Amicus has set an initial WAC price of $315,000. 
• The FDA approved Alnylam Pharmaceuticals’ Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. The drug has a Breakthrough Therapy Priority Designation. This is the first FDA-approved treatment for patients with polyneuropathy caused by hATTR, a rare, debilitating and often fatal genetic disease characterized by the accumulation of abnormal amyloid protein in peripheral nerves, the heart and other organs. It is also the first FDA approval of a new class of drugs called small interfering ribonucleic acid (siRNA) treatment.
• The FDA approved Shire’s lanadelumab (Takhzyro) on 8/23/18 for the treatment of types I and II hereditary angioedema. Lanadelumab has Orphan Drug and Breakthrough Therapy Priority Designations.
• The FDA approved Bayer's antihemophilic factor PEGylated-aucl (Jivi) on 8/30/18 for the routine prophylactic treatment of hemophilia A.
• Tetraphase Pharmaceuticals’ eravacycline (Xerava) was approved for the treatment of complicated intra-abdominal infections. The drug is forecast to be available by the end of October. Eravacycline has a broad indication and can potentially be used for empiric therapy. The drug will be priced at $175 to $250 per day.
• The FDA approved Merck's doravirine (Pifeltro) and doravirine, lamivudine and tenofovir disoproxil fumarate (Delstrigo) for the treatment of HIV on 8/31/18
• The FDA followed the recommendation of its subcommittees and rejected Mallinckrodt’s stannsoporfin.
• The FDA rejected Ionis Pharmaceuticals’ volanesorsen on 8/27/18 although an FDA advisory committee recommended approval in early May.
• An FDA Advisory Committee voted to recommend approval of Paratek Pharmaceuticals’ IV and oral omadacycline to treat skin and skin structure infections and for its use in CAP. The drug has Fast Track and a Qualified Infectious Disease Product Priority Designations with a PDUFA date of October 4, 2018.
• The FDA granted Motif Bio’s iclaprim a priority review. PDUFA Date: Feb 13, 2019. The drug has Fast Track and Qualified Infectious Disease Product Priority Designations. Iclaprim Is given as a 2-hour infusion Q12 hr. In four Phase III skin infection trials, the most common ADRs were headache, nausea, vomiting, fatigue. It is the only DHFR-Inhibitor in our investigational knowledgebase. Its indication is acute bacterial skin and skin structure infections and hospital acquired bacterial pneumonia.
• Alexion Pharmaceuticals’ ravulizumab was granted a priority review by the FDA and given a PDUFA date of 2/18/2019. Ravulizumab has orphan drug status in the US and EU.
• Ravulizumab, was granted a priority review by the FDA and given a PDUFA date of 2/18/2019. Ravulizumab has orphan drug status in the US and EU.
• The FDA rejected dasotraline on 8/31/18 and requested additional data on efficacy and tolerability of dasotraline for the treatment of ADHD.
  

Noteworthy updates for late stage drugs include:

• Novo announced that in a Phase III trial, patients with type 2 diabetes and moderate renal impairment, treated with oral semaglutide lowered their HbA1c 1.1% and lost 3.7 kg compared to a HbA1c decrease of 0.1% and 1.1 kg loss with placebo.
• Tonix reported that low dose cyclobenzaprine was no better than placebo in a Phase III trial in an interim analysis, so the trial was discontinued. A subgroup analysis suggested a benefit in patients with PTSD less than 9 years duration.
• Jiangsu announced that in a Phase II trial, treatment with apatinib monotherapy demonstrated an ORR of 41.4% and a PFS of 5.1 months in patients with platinum-resistant ovarian cancer.
• Bapineuzumab (Pfizer and Johnson & Johnson) In four Phase III trials, did not demonstrate efficacy in patients with mild to moderate Alzheimer's disease. Pfizer discontinued development of bapineuzumab for Alzheimer's disease due to a lack of efficacy.
• The Dengue vaccine from Sanofi in two Phase III pediatric trials demonstrated vaccine efficacy of 56.5% and 60.8%, respectively, against symptomatic virologically confirmed dengue. The vaccine appeared to be more effective in older children possibly due to higher baseline seropositivity and potentially a more broadly protective immune response.
• Gilead announced that two Phase IIb simtuzumab NASH trials, were stopped early when interim data from 197 patients that had completed 96 weeks of therapy did not demonstrate an improvement in hepatic collagen content, bridging fibrosis or cirrhosis.
• Noimmune has submitted an MMA in the EU for emapalumab.
• Preliminary results from the first 10 patients in the Phase III CardiAMP-HF Trial, suggested that a six-month open label treatment with CardiAMP improved Six Minute Walk Distance by 48 meters in patients with ischemic cardiomyopathy, LVEF 20-40%.
• Sierra Oncology bought momelotinib from Gilead Sciences in August 2018.
• Neurocrine announced that in an extension of BIPARK I trial, opicapone (adjunct to levodopa therapy in patients with Parkinson's Disease) in patients experiencing end-of-dose motor fluctuations, compared to entacapone, reduced off periods by 65 minutes and 39 minutes and increased on periods without troublesome dyskinesia by 43 minutes and 46 minutes.
• Acacia Pharma announced that in a 560-patient trial, 31% of patients treated with amisulpride had no emesis or need of rescue medications compared to 21% with placebo in patients at low-to-moderate risk for PONV who had not received any prior prophylaxis.

You can access up-to-date information on all drugs in the late stages of development with the Prescribe Right Pharmaceutical Pipeline Tracker.​