January 2020 decisions expected from the FDA

At Prime Therapeutics (Prime), we’ve positioned ourselves to best 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).

1/1/2020: Palforzia™ (AR101)

The FDA will decide on Aimmun Therapeutics’ Palforzia, an oral treatment for peanut allergy in children and adolescents ages 4 to 17 years old. Palforzia is an oral powder in pull-apart capsules or foil-laminate sachets that has gradual dosing from 0.5-300 mg/day. The initial dose and first dose of each dose-escalation level must be administered in a facility equipped to treat systemic allergic reactions. The allergenic Products Advisory Committee (APAC) voted 7 to 2 that the efficacy data and 8 to 1 that the safety data, in conjunction with additional safeguards, are adequate to support the use of Palforzia.1 The Institute for Clinical and Economic Review’s (ICER) final draft was published July 2019; its review voted 12 to 4 that evidence was inadequate to demonstrate a superior net health benefit of Palforzia compared to strict peanut avoidance. Adverse reactions with Palforzia were substantially higher than those observed in the placebo group. Most common reactions were gastrointestinal pain, nausea and vomiting (52%) and systemic allergic reactions (14.2% versus 3.2% placebo).2 Palforzia will be distributed through specialty pharmacies and purposefully designed packaging so patients only receive their appropriate doses.

1/20/2020: Rybelsus® (semaglutide, oral tablet)

The FDA is reviewing Novo Nordisk’s Rybelsus for the reduction of major adverse cardiovascular events (MACE) in adults with type 2 diabetes. Rybelsus is the first oral glucagon-like peptide-1 (GLP-1) available in tablet formulation. It is currently approved as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes.3 Other GLP-1 include AstraZeneca’s Byetta® (exenatide), Novo Nordisk’s Victoza® (liragluide), Sanofi’s Adlyxin® (lixisenatide), Merck’s Trulicity® (dulaglutide) and Novo Nordisk’s Ozempic® (semaglutide).

1/23/2020: Tazemetostat

Epizyme’s Tazemetostat is being reviewed for accelerated approval for metastatic or locally advanced epithelioid sarcoma not eligible for curative surgery. Tazemetostat is a first-in-class EZH2 inhibitor. The recommended dose is 800 mg by mouth twice a day. Epizyme’s NDA submission is based primarily on data from the 62-patient epithelioid sarcoma cohort ongoing Phase 2 study of Tazemetostat which demonstrated clinically meaningful and durable responses. To support full approval of Tazemetostat for epithelioid sarcoma, Epizyme will initiate a global confirmatory trial. The company plans to conduct a 1:1 randomized, controlled clinical trial in the front-line treatment setting comparing Tazemetostat in combination with doxorubicin versus placebo plus doxorubicin in approximately 150 patients. The primary efficacy endpoint will be progression-free survival, and secondary efficacy endpoints will include overall survival, disease control rate, overall response rate and duration of response.4 Similar products by indication include Novartis’ Votrient® (pazopanib), Eli Lilly’s Gemzar® (gemcitabine), Loxo Oncology’s Vitrakvi® (larotrectinib), Merck’s Keytruda® (pembrolizumab) or ifosfamide.

1/24/2020: Dificid™ (fidaxomicin)

The FDA is reviewing Merck’s Dificid oral suspension formulation of the macrolide antibiotic for treatment of Clostridium difficile-associated diarrhea (CDAD). Merck has also submitted an NDA for Dificid for a new indication for use of tablets and oral suspension for the treatment of Clostridium difficile infections in children aged six months or older. Both applications have received priority review from the FDA. Dificid is not effective for treatment of other types of infections due to minimal systemic absorption of fidaxomicin.5 If approved it will join the standard treatments for CDAD which include oral metronidazole and oral vancomycin.

1/28/2020: Rykindo™ (risperidone, extended-release injection)

Luye Pharma Group’s Rykindo is being reviewed by the FDA for treatment of schizophrenia and bipolar disorder. Rykindo is an extended-release formulation using Luye’s microsphere technology for intramuscular injection once every two weeks. Luye submitted Rykindo under the 505(b)(2) pathway using Janssen Pharmaceuticals’ Risperdal Consta® (risperidone) as a reference drug. The dosages of Rykindo to be marketed are 12.5 mg, 25 mg, 37.5 mg and 50 mg of risperidone per vial.6 Risperidone is currently available as a generic tablet and injectable.

The drug pipeline is full of new drugs that may help members feel better and live well. Prime drug pipeline tracking helps keep you on top of drug trends, because it’s easier to manage change when you can see it coming.

---

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.

---

References

1. http://ir.aimmune.com/news-releases/news-release-details/fda-allergenic-products-advisory-committee-votes-support-use
2. https://icer-review.org/wp-content/uploads/2019/07/ICER_PeanutAllergy_RAAG_071019.pdf
3. https://www.novonordisk-us.com/media/news-releases.html?122973
4. https://epizyme.gcs-web.com/news-releases/news-release-details/epizyme-announces-fda-filing-acceptance-new-drug-application-and
5. https://investors.merck.com/news/press-release-details/2019/FDA-Accepts-Two-Applications-for-Mercks-DIFICID-fidaxomicin-to-Treat-Children-Aged-Six-Months-Up-to-18-years-with-Clostridium-difficile-Infections/default.aspx
6. https://clarivate.com/cortellis/wp-content/uploads/sites/4/2019/06/Regulatory-BW-article-Luye-files-FDA-NDA-for-China-developed-Rykindo.pdf