July 2022 decisions expected from the FDA

June 20, 2022
At Prime Therapeutics (Prime), we’ve positioned ourselves to best prepare our clients to manage new drugs. 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).

Drug pipeline for July 2022:

 7/3/2022: olipudase alfa
The FDA is reviewing Sanofi/Genzyme’s olipudase alfa as an enzyme replacement therapy for long-term treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick disease type A and type B) in pediatric and adult patients. ASMD is a rare, progressive, and potentially life-threatening disease that results from a deficient activity of the enzyme acid sphingomyelinase (ASM), which is found in special compartments within cells called lysosomes and is required to breakdown lipids called sphingomyelin. If ASM is absent or not functioning as it should, sphingomyelin cannot be metabolized properly and accumulates within cells, eventually causing cell death and the malfunction of major organ systems. The deficiency of the lysosomal enzyme ASM is due to disease-causing variants in the sphingomyelin phosphodiesterase 1 gene (SMPD1). The estimated prevalence of ASMD is approximately 2,000 patients in the United States, Europe, and Japan.1 Olipudase alfa is seeking approval based on the Phase 3 ASCEND trial that demonstrated significantly improved lung function and significantly reduced spleen volume for olipudase alfa-treated patients at 52 weeks. Additionally, olipudase alfa’s data remained consistent throughout the 6.5-year outcomes for five adult patients with ASMD and 2-year outcomes in 20 pediatric patients, as well as the open-label extension from the Phase 3 ASCEND trial in adults. Results showed the percent predicted lung function (DLco) mean increase was 55.3%, spleen volume mean decrease was 59.5%, and liver volume mean decrease was 43.7% compared to baseline of original study.1 Currently there are no similar products available for this indication that are approved by the FDA.

 7/12/2022: tislelizumab (BGB-A317)
BeiGene and Novartis’ tislelizumab (BGB-A317) is being evaluated by the FDA for treatment of patients with unresectable recurrent locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic therapy. ESCC is the most common type of esophageal cancer globally and the sixth leading cause of cancer-related death worldwide.2 There are approximately 17,000 people living with ESCC in the United States and in 2021, and it is estimated that there will be 19,000 new esophageal cancer cases diagnosed and more than 15,000 deaths from esophageal cancer. Tislelizumab is seeking approval based on Phase 3 RATIONALE 302 trial which demonstrated a 30% reduction in the risk of death and extended median overall survival by 2.3 months compared to chemotherapy in people with unresectable recurrent locally advanced or metastatic ESCC who had received prior systemic therapy.2 Similar products include Merck’s Keytruda® (pembrolizumab).

 7/14/2022: vutrisiran
The FDA is reviewing Alnylam Pharmaceuticals’ vutrisiran for treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Vutrisiran is a second-generation formulation of Onpattro® (patisiran) which is an RNA interference (RNAi) therapy that blocks production of wild-type and variant transthyretin protein. Vutrisiran’s primary advantage over Onpattro is its dosing regimen. Patients taking Onpattro must undergo an 80-minute infusion every three weeks. Vutrisiran, on the other hand, is a subcutaneous injection that is administered by a health care professional every three months. Vutrisiran is seeking approval based on HELIOS-A, a Phase 3 open-label study which evaluated the efficacy and safety of vutrisiran compared to the Onpattro IV infusion. The primary endpoint was the change from baseline in mNIS+7 score at nine months and vutrisiran was found to be non-inferior.3 Similar products include Alnylam’s Onpattro® (patisiran) and Akcea Therapeutics’ Tegsedi® (inotersen).

7/29/2022: roflumilast cream (ARQ-151)
Arcutis Biotherapeutics’ roflumilast cream (ARQ-151) is being evaluated by the FDA for the treatment of mild-to-severe plaque psoriasis. Rolumilast cream is a once-daily highly potent, selective inhibitor of phosphodiesterase type 4 (PDE4). Roflumilast cream’s submission is based on two Phase 3 studies, DERMIS-1 and DERMIS-2 that demonstrated an Investigator Global Assessment success rate of 46.4% compared to 6.1% in vehicle treated group (DERMIS-1) and 37.5% compared to 6.9% with vehicle (DERMIS-2) respectively.4 Roflumilast is currently approved in oral tablet form indicated for chronic obstructive pulmonary disease as AstraZeneca’s Daliresp® (roflumilast). Similar products include a variety of topical treatments including corticosteroids, emollients, vitamin D analogs and generic retinoids.

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.


  1. https://www.sanofi.com/en/media-room/press-releases/2022/2022-02-09-18-00-00-2382138
  2. https://www.novartis.com/news/media-releases/novartis-announces-first-fda-filing-acceptance-anti-pd-1-antibody-tislelizumab-people-esophageal-cancer
  3. https://www.businesswire.com/news/home/20210624005230/en/Alnylam-Announces-U.S.-Food-and-Drug-Administration-Acceptance-of-New-Drug-Application-for-Investigational-Vutrisiran-for-the-Treatment-of-the-Polyneuropathy-of-Hereditary-ATTR-Amyloidosis
  4. https://www.dermatologytimes.com/view/fda-accepts-nda-for-roflumilast-cream


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