May 2022 decisions expected from the FDA
Your monthly synopsis of new drugs expecting FDA decisions in May 2022April 13, 2022
5/2022: Cuprior® (trientine tetrahydrochloride (TETA 4HCl))
The FDA is reviewing Orphalan’s Cuprior for first-line treatment of Wilson’s disease, a rare inherited disorder of copper transport primarily affecting the liver and brain, affecting about one in every 30,000 people worldwide. Cuprior is proposed as an alternative copper chelating agent to d-Penicillamine, the only approved first-line treatment of Wilson’s Disease for the last 70 years, to which about a third of patients develop intolerance. Cuprior is seeking approval based on the Phase 3 CHELATE clinical trial that met its primary efficacy endpoint by demonstrating that Cuprior was non-inferior to d-Penicillamine as measured by copper speciation evaluation of non-ceruloplasmin copper (NCC).1 Similar products include Valeant Pharmaceuticals’ Syprine® (trientine hydrochloride) which is indicated in the treatment of patients with Wilson’s disease who are intolerant of penicillamine.
The FDA is reviewing Phathom Pharmaceuticals’ vonprazan for two indications. First as a dual therapy regimen in combination with the antibiotic amoxicillin for treatment of Helicobacter pylori (H. pylori) infection in adults as well as triple therapy regimen in combination with the two antibiotics (amoxicillin and clarithromycin) for treatment of Helicobacter pylori infection in adults. Vonoprazan is an oral potassium-competitive acid blocker (P-CAB). It is suggested to have a more rapid onset of action and reduce hepatic toxicity compared with conventional proton pump inhibitors. Vonoprazan’s application is based on both regimens successfully meeting their primary endpoints of being non-inferior compared to lansoprazole combination regimens. According to the Phase 3 PHALCON-HP trial vonoprazan was non-inferior to lansoprazole in H. pylori eradication in combination with clarithromycin and amoxicillin (84.7 versus 78.8%) and in combination with amoxicillin alone (78.5% versus 78.8%).2 Similar products include generic lansoprazole.
5/12/2022: edaravone, oral (MT-1186)
Mitsubishi Tanabe Pharma’s edaravone is seeking priority approval from the FDA for treatment of amyotrophic lateral sclerosis (ALS). Edaravone is an oral formulation of the free radical-scavenging nootropic and neuroprotective agent. Edaravone is already approved as intravenous (IV) Radicava®. Edaravone oral formulations application includes several Phase 1 clinical pharmacology studies comparing the oral suspension to the IV formulation. Given that Radicava IV’s dosing regimen requires hour-long infusions and frequent visits to the clinic (14 consecutive days in the first treatment cycle and 10 consecutive days in the following cycles), an oral formulation could reduce patient burden and health care resources. Additionally, results from an open-label Phase 3 study that evaluated the safety and tolerability of edaravone oral suspension which demonstrated a favorable safety profile.3
5/24/2022: Ycanth® (cantharidin 0.7% topical solution)
Verrica Pharmaceuticals’ Ycanth is being reviewed by the FDA as a drug-device combination for topical treatment of molluscum contagiosum. Molluscum contagiosum is a highly contagious viral skin infection that affects close to 6 million people in the United States each year. Cantharidin is currently available as a compounded agent. Verrica Pharmaceuticals is seeking FDA approval based on the Phase 3 CAMP trial that demonstrated Ycanth to be statistically significant when compared to placebo. Efficacy was measured by the percentage of subjects with complete clearance of lesions in each location (head, chest, back, groin, upper and lower extremities) by day 84 compared to vehicle.4 When used on patient’s head/neck, 81.8% of Ycanth patients experienced complete clearance compared to 39.6% with vehicle. Overall, 50% of patients using Ycanth experienced complete clearance compared with 15.6% response with use of the vehicle. Verrica Pharmaceuticals intends to conduct additional clinical trials later this year to evaluate Ycanth for treatment of common warts and external genital warts. Similar products include cryotherapy, cantharidin compounded solution, and Allergan Pharmaceuticals’ Condylox® (podofilox).
5/26/2022: tapinarof (DMVT-505)
Dermavant (Roivant)’s tapinarof is seeking approval by the FDA for treatment of mild, moderate and severe plaque psoriasis in adults. Tapinarof is a therapeutic aryl hydrocarbon receptor modulating agent (TAMA) formulated as a topical cream for once daily use. Tapinarof is seeking approval based on two Phase 3 clinical trials PSOARING-1 and PSOARING-2 which compared tapinarof cream 1% to vehicle. Primary efficacy was defined as Physician Global Assessment (PGA) score of 0 or 1 (clear or almost clear) and a decrease of at least 2 points of the 5-point PGA scale at 12 weeks. In PSOARING 1, a PGA response was recorded in 35.4% of those in the tapinarof treatment group, compared with 6% of those in the vehicle group, while 40.2% of the PSOARING 2 treatment group and 6.3% of the PSOARING 2 vehicle group had the same response at week 12.5 Similar products include Galderma Laboratories’ Clobex® (clobetasol propionate 0.05%), Perrigo’s Luxiq® (betamethasone valerate 0.12%),Bausch Health’s Vanos® (fluocinonide cream), vitamin D modulators, retinoids, fixed-dose corticosteroid/vitamin D modulator combinations and off-label use of topical calcineurin inhibitors.
5/29/2022: Miglustat™ (with cipaglucosidase alfa)
The FDA is reviewing Amicus Therapeutics’ Miglustat in combination with cipaglucosidase alfa to treat patients with Pompe disease. Pompe is a very rare disease, with an estimated incidence of 1 in 20,000 to 100,000 births and a worldwide prevalence of 5,000 to 10,000 individuals. The two-component therapy for Pompe disease is comprised of the intravenous enzyme-replacement therapy (ERT) cipaglucosidase alfa co-administered with the oral molecular chaperone Miglustat. Cipaglucosidase alfa, an enhanced version of the standard of care, Sanofi’s Lumizyme® (alglucosidase alfa), contains modifications in the protein backbone and in the surface glycosylation pattern to reduce immunogenicity and to enhance targeting to key affected muscles. Miglustat is applying for approval based on the Phase 3 PROPEL study of its combination therapy, AT-GAA, for the treatment of late onset Pompe disease (LOPD). While the results were good, they were not great. Miglustat did not meet the primary endpoint of the study of the 6-minute walk test (6MWT) when comparing the group given AT-GAA with the ERT-naive group receiving the current standard of care, alglucosidase alfa. Results from the ERT switch portion of the study were much more encouraging, although the overall 6MWT results still weren’t statistically significant.
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