May 14, 2020
June 2020 decisions expected from the FDA
Your snapshot of new drugs expecting an FDA decision in June 2020.
Prime works hard to prepare our clients to manage the new drugs that come on the market. Our clinical and trade relations teams keep a keen eye on drugs likely to be approved by the U.S. Food and Drug Administration (FDA).
6/2/2020: FMX103 minocycline foam 1.5%
The U.S. Food and Drug Administration (FDA) is reviewing Foamix Pharmaceuticals FMX103 minocycline foam 1.5% for the treatment of severe papulopustular rosacea in patients 18 years and older. Foamix Pharmaceuticals is seeking approval based on two Phase 3 studies with 1,522 subjects that compared FMX103 to vehicle foam. FMX103 demonstrated statistically significant benefit compared to vehicle foam for reduction of inflammatory lesion counts.1 Similar products include Foamix Pharmaceuticals’ Amzeeq® (minocycline topical foam, 4%) and oral minocycline generics.
The FDA has granted Priority Review to Shinyaku’s viltolarsen for the treatment of Duchenne muscular dystrophy (DMD). Viltolarsen is an infusion that skips exon 53 when making mature mRNA. Due to vitolarsen’s specificity to exon 53, DMD patients must have a confirmed exon 53 mutation which represents approximately 8% of all DMD patients.2 Clinical benefits have not been established for this patient population. Shinyaku is seeking accelerated approval based on surrogate endpoints from a Phase 2 clinical trial that compared viltolarsen against placebo in 16 boys with DMD who were still able to walk after six months. Trial results demonstrated that viltolarsen restored dystrophin production in the patient’s muscles after 20 to 24 weeks after infusion; however, these endpoints have not been proven to slow disease progression for DMD. An open-label extension of this clinical is ongoing to continue monitoring the effects of viltolarsen, once weekly infusions for an additional 144 weeks. The trial is due to conclude December 2020.3 Viltolarsen is currently recruiting for a Phase 3 study, RACER53, to further evaluate safety and efficacy. Viltolarsen was granted Rare Pediatric Disease, Orphan Drug, and Fast Track designation by the FDA. Similar products include Sarepta’s Vyondys 53® (golodirsen).
6/19/2020: Contepo™ (fosfomycin for injection)
The FDA has granted Priority Review of Nabriva Therapeutics’ Contepo™ (fosfomycin for injection) for complicated urinary tract infections (cUTI). Contepo is a broad-spectrum antibiotic that has gram-negative and gram-positive activity including activity against most contemporary multi-drug bacteria. IV Fosfomycin has been approved for several indications and utilized for over 45 years outside the United States to treat a variety of infections, including cUTIs and other serious bacterial infections. Nabriva is seeking approval based on Phase 2/3 clinical trial, ZEUS, which demonstrated non-inferiority to piperacillin/tazobactam in patients with cUTI.4 In April 2019, Nabriva received a complete response letter (CRL) from the FDA due to issues related to facility inspections and manufacturing deficiencies. In January 2020, Nabriva resubmitted its application for review. Similar products include Pfizer’s Zosyn® (piperacillin/tazobactam).
6/19/2020: Gimoti™ (metoclopramide nasal spray)
The FDA is reviewing Evoke Pharma’s Gimoti™ (metoclopramide nasal spray) for relief of symptoms in adult women with acute and recurrent diabetic gastroparesis. Evoke pharma submitted Gimoti under a 505(b)(2) pathway using Ani Pharmaceuticals’ Reglan® (metoclopramide) tablets as its reference drug. Evoke Pharma states that Gimoti is designed to deliver metoclopramide faster than tablets do; however, studies have not demonstrated this claim. In April 2019, the FDA issued a CRL expressing concerns about Gimoti’s chemistry, clinical and pharmacology information included in the application. In addition, the CRL included concerns regarding product and device quality. The FDA requested additional data to explain PK variability and batch droplet size distribution.5 In January 2020, Evoke Pharma resubmitted Gimoti application to the FDA. Similar products include Ani Pharmaceuticals’ Reglan® (metoclopramide).
Gilead’s filgotinib has been granted Priority Review by the FDA for treatment of rheumatoid arthritis (RA). Filgotinib is an oral selective JAK1 inhibitor. Gilead submitted filgotinib based on results from a Phase 3 clinical trial, FINCH 1, that demonstrated filgotinib 100mg and 200mg achieved a 20% response (ACR20) as defined by the American College of Rheumatology compared to placebo at week 12. Additionally, filgotinib demonstrated a statistically significant reduction in the Health Assessment Questionnaire Disability Index (HAQ-DI) at week 12 compared with those receiving placebo.6 Gilead is further exploring filgotinib in the treatment of psoriatic arthritis, ankylosing spondylitis, Crohn’s disease and ulcerative colitis. Gilead touts filgotinib’s ability to avoid safety problems that Pfizer’s Xeljanz® (upadacitinib) and Eli Lilly’s Olumiant® (baricitinib) have but safety is uncertain. AbbVie’s Rinvoq® (upadacitinib), shares filgotinib’s specific JAK1 mechanism of action and received a black box warning.7 Other similar products include Pfizer’s Xeljanz® (upadacitinib) and Eli Lilly’s Olumiant® (baricitinib).
6/25/2020: Fintepla™ (fenfluramine, ZX008)
Zogenix’s Fintepla is being reviewed by the FDA as a low dose of the serotenergic agent for treatment of seizures associated with Dravet syndrome. Fintepla is an oral solution that cut convulsive seizures by 63.6% vs placebo in patients over 6 years of age with Dravet. In patients under 6 years of age patients showed a decrease in convulsive seizures by 75.5%.8 In April 2019, Zogenix was issued a refuse to file letter from the FDA citing a lack of certain non-clinical studies to assess the chronic administration of Fintepla and due to an incorrect dataset. In September 2019, Zogenix resubmitted their application. Similar products include Lundbeck’s Onfi® (clobazam), Greenwich Biosciences’ Epidiolex® (cannabidiol oral solution).
6/25/2020: obeticholic acid (OCA)
Intercept Pharmaceuticals’ obeticholic acid has been granted Priority Review by the FDA for treatment of fibrosis due to nonalcoholic steatophepatitis (NASH). Obeticholic acid was previously approved as Ocaliva® for primary biliary cholangitis. Obeticholic acid’s regulatory submissions are based on REGENERATE, a Phase 3 study which demonstrated OCA 25mg, improved liver scarring with no worsening of NASH at 23.1% versus 11.9% in patients with stage 2 or 3 fibrosis due to NASH.9 Obeticholic acid was unable to demonstrate a statistically significant number of patients who achieved NASH resolution compared to placebo; therefore, failing to meet its second endpoint. The FDA scheduled an advisory committee hearing to review Intercepts application in June 2020. If approved, obeticholic acid will be launched as a 25mg tablet under a different trade name for NASH. There are currently no FDA approved therapies for the treatment of NASH.
6/26/2020: HTX-011 (bupivacaine and meloxicam)
Heron Therapeutics is requesting the FDA review its HTX-011 (bupivacaine and meloxicam) combination product for management of postoperative pain. This injection uses Heron's Biochronomer delivery technology for local administration into the surgical site. HTX-011 has received Fast Track, Breakthrough Therapy, and Priority Review from the FDA. Phase 3 studies demonstrated significant reduction in pain and opioid use through 72 hours compared to bupivacaine solution, which is the current standard-of-care local anesthetic for postoperative pain control.10 In April 2019, HTX-011 received a complete response letter (CRL) relating to chemistry, manufacturing and controls and non-clinical information. Heron resubmitted an NDA to the FDA in September 2019. Similar products include Pfizer’s Marcaine® (bupivacaine hydrochloride), and Boehringer Ingelheim’s Mobic® (meloxicam).
6/26/2020: Mycapssa™ (octreotide)
Chiasma’s Mycapssa is being reviewed by the FDA as an oral capsule formulation of the octreotide using Chiasma's Transient Permeability Enhancer technology for maintenance treatment of adults with acromegaly. Chiasma is seeking approval based on two Phase 3 clinical trials: CHIASMA OPTIMAL, and MPOWERED. CHIASMA OPTIMAL trial was conducted under a special protocol assessment agreement with the FDA to evaluate Mycapssa for the maintenance treatment of adults with acromegaly. It achieved patients control of biochemistry (IGF-1 ≤ 1.0 x ULN). MPOWERED results are on track for the fourth quarter of 2020.11 In April 2016, the FDA issued a CRL for Mycapssa. In January 2020, Chiasma resubmitted its application for Mycapssa. If approved Chiasma has plans in place to launch Mycapssa fourth quarter of 2020. Similar products include Novartis’ Sandostatin® (octreotide) and its generics.
The FDA is reviewing Viela Bio’s inebilizumab for Neuromyelitis Optica Spectrum Disorder (NMOSD). There are approximately 10,000 NMOSD patients in the United States. Inebilizumab met its primary efficacy endpoint with a 77% reduction in risk of developing an NMOSD attack in AQP4-IgG seropositive patients when compared to placebo after 28 weeks of treatment and 89% reduction in risk at end of controlled trial (compared with 58% in placebo group). Additionally, inebilizumab demonstrated 73% reduction in attack risk for total patient population enrolled compared to placebo.12 Viela Bio is planning on additional clinical trials to investigate inebilizumab in myasthenia gravis and IgG4-related diseases in mid-2020. Similar products include Alexion Pharmaceuticals Soliris® (eculizumab).
While the information in this newsletter is from sources we believe to be reliable, we do not warrant that the information in this document is free from error. Use it only as a guide. Statements regarding drugs or manufacturers are not intended as promotion; those statements should not be used to make assumptions about formulary status. Each trademarked drug name is the property of its respective owner.
About Prime Therapeutics
Prime Therapeutics LLC (Prime) makes health care work better by helping people get the medicine they need to feel better and live well. Prime provides total drug management solutions for health plans, employers, and government programs including Medicare and Medicaid. The company processes claims and offers clinical services for people with complex medical conditions. Prime serves more than 28 million people. It is collectively owned by 18 Blue Cross and Blue Shield Plans, subsidiaries or affiliates of those plans.
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