September FDA Approvals

For the second month in a row the FDA pushed through five new drug approvals. This was accomplished by a surge of activity during the last week of the month for cancer and migraine drugs.  The FDA rejected one drug, and another failed to produce its expected results. There were updates to nine drugs with PDUFA Dates and Priority Designations (potential budget busters). Read on for the details.
APPROVALS

• Two FDA approved the second and third CGRP-Receptor Antagonists in September. They join erenumab (Amiovig, Amgen/Novartis) as new options for migraine prophylaxis. All three drugs have a WAC of $575 per syringe (6,900 annually). All three drugs also have a discount program that covers the copay for patients with commercial insurance.  
  • Teva’s fremanezumab (Ajovy) was approved for the prevention of migraines, on 9/14/18, two days ahead of its PDUFA date. Fremanezumab will be available in pre-filled syringes dosed as 225mg monthly or 675mg (3 x 225mg doses) every 3 months. The drug had a Fast Track Priority Designation.
  • Lilly’s galcanezumab (Emgality) was approved on 9/28/2018, 2 days ahead of its PDUFA Date, as a once-monthly, self-administered, subcutaneous injection for the preventive treatment of migraine in adults. The drug had Fast Track Priority Designation. 
• The FDA approved Verastem’s duvelisib (Copiktra) on 9/24/18 ahead of the Oct 5, 2018 PDUFA Date, for the treatment of patients with relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) or relapsed/refractory follicular lymphoma. Duvelisib has orphan drug and fast track Priority Designations.
• The FDA approved Pfizer’s dacomitinib (Vizimpro) on 9/27/2018, 3 days ahead of its PDUFA Date, as first-line treatment of patients with metastatic non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 19 deletion or exon 21 L858R substitution mutations, as detected by an FDA-approved test. WAC was set at $12,400 for one month’s treatment.
• The FDA approved Regeneron Pharmaceuticals’ and Sanofi Aventis’ cemiplimab (Libtayo) on 9/28/18, one month ahead of its PDUFA Date, for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation. The WAC for a three-week treatment cycle is $9,100. The drug has a Breakthrough Therapy Priority Designation.

REJECTIONS & DISCONTINUATIONS

• The FDA rejected Sunovion Pharmaceuticals’ dasotraline on 8/31/18 and requested additional data on efficacy and tolerability of dasotraline for the treatment of ADHD.
• After failure in the CONCERTO and ARPEGGIO trials, Teva stopped development of laquinimod for multiple sclerosis. After the failure of a Phase II Huntington's disease trial, Teva ceased all development projects for laquinimod and returned all rights to Active Biotech.

UPDATES TO PRODUCTS WITH PDUFA DATES AND PRIORITY DESIGNATIONS (Potential budget busters)

• In a 1,064 patient, Phase III, CAPSTONE-1 trial, a single dose of baloxavir marboxil (Genentech, Shionogi) reduced the time to alleviation of symptoms of influenza infection by 26.5 hours compared to placebo and was similar to 5 days of oseltamivir. Baloxavir marboxil also reduced viral load more rapidly than oseltamivir or placebo. The drug’s PDUFA date is December 24, 2018, but has no Priority Designations at present. In the Phase III, CAPSTONE-1 trial, nausea was less common with a single dose of baloxavir marboxil compared to 5 days of oseltamivir.
• A phase II study showed a decrease in tumor volume and 4-5-month progression-free survival with talazoparib in resistant BRCA1/2 mutation breast cancer patients. Pfizer announced that in a 20 patient, Phase II trial of patients with BRCA+ breast cancer, patients received 6 months of talazoparib prior to surgery. Before surgery, 63% of patients had a residual cancer burden score of RCB0 or RCB1, which indicates the same positive expected outcome. In the 287 patient, Phase III, EMBRACA trial, patients treated with talazoparib had a PFS of 8.6 months compared to 5.6 months with standard chemotherapy and an ORR of 63% vs 27% in patients with advanced breast cancer who carry the BRCA1/2 mutations. PDUFA Date: December 15, 2018. No Priority Designations.
• Jazz announced that in patients with narcolepsy or obstructive sleep apnea, solriamfetol-treated patients in Phase III Trials had an improvement in wakefulness and a decrease in sleepiness compared to placebo. Jazz also announced a subset of 280 patients participated in a 2-week withdrawal trial, where patients switched from solriamfetol to placebo had a decrease in wakefulness and an increase in sleepiness. PDUFA date Dec 20, 2018, Orphan Drug Priority Designation. Jazz filed an NDA for solriamfetol. The drug has a PDUFA Date of Dec 20, 2018, and an Orphan Drug Priority Designation.
• Acacia Pharma’s amisulpride (Barhemsys) for post-operative nausea and vomiting, has a PDUFA date of 10/5/18.
• Regeneron announced that in a Phase I trial, treatment with cemiplimab (Libtayo) monotherapy resulted in an overall response rate of 40% and 18% when combined with radiotherapy in patients with relapsed/refractory non-small cell lung cancer. PDUFA Date: Oct 28, 2018 with a Breakthrough Therapy Priority Designation.
• Pfizer is enrolling patients in the Phase III CROWN trial (NCT03052608) comparing lorlatinib with crizotinib as a first-line therapy for ALK-positive NSCLC. Lorlatinib has a PDUFA Date of November 30, 2018 with Orphan Drug and Breakthrough Priority Designations.
• Govitecan is the active metabolite of irinotecan. It is bound to sacituzumab, a trophoblastic antigen-2 antibody, a receptor found on many tumors. Immunomedics is seeking approval for the treatment of metastatic triple-negative breast cancer in patients that have previously received at least two prior therapies for metastatic disease. Immunogenics has an ongoing Phase III trial evaluating sacituzumab govitecan in the treatment of triple negative breast cancer. The drug’s PDUFA Date is Jan 18, 2019.

​31 citations were added to our knowledgebase bringing the total to 811. Each citation is accessible from the single drug look-up monograph via a URL link to the publication. There are now 612 investigational drugs in the Knowledgebase.

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