The Latest Developments in Drug Approvals
Each quarter, the RemedyOne Clinical team curates a drug pipeline report for our clients that shares insight into specialty and traditional drugs in development that are expected to launch within the next twelve months, with a focus on medications currently in Phase III studies. The report is a helpful tool for pharmacy teams to stay updated on the latest drugs to market and to plan pharmacy benefits.
This article will provide an update on the drugs in the previous quarter’s pipeline (Q3/July 2021) that were awaiting FDA approval and have now gained approval for their indications. Our team has summarized background information regarding the drug, disease state and how it may play out with other therapies for these newly approved drugs.
Aduhelm (aducanumab), by Eisai/Biogen
Approved June 2021 for the treatment of Alzheimer’s disease in patients with mild cognitive impairment or mild dementia stage of disease. Alzheimer’s affects nearly 6 million geriatric Americans. It is a disease characterized by plaque accumulation of amyloid beta in the brain. This accumulation leads to issues with memory recall, thinking, learning, and daily life activities. Currently, the treatment of Alzheimer’s entails symptom management. Aduhelm estimated annual cost is $56,000 based on the average weight of an adult patient (80kg). The studies on Aduhelm are conflicting, including conclusions that found either zero or slight efficacy. The FDA decided to approve Aduhelm based on a surrogate endpoint instead of actual clinical benefit in improved cognition. Aduhelm was approved despite conflicting studies and a near unanimous vote by the FDA’s neurological drugs advisory panel not to approve the drug. Many payers and employer groups have been reluctant to cover this drug and are waiting for a coverage determination by CMS (which may not come until early next year) to help guide their decision.
Kerendia (finerenone), by Bayer
Approved July 2021 to reduce the risk of sustained estimated glomerular filtration rate (eGFR), end stage kidney disease (ESKD), cardiovascular (CV) death, non-fatal myocardial infarction (MI), and hospitalization for heart failure (HF) in adult patients with chronic kidney disease (CKD) associated with Type 2 Diabetes (T2D). There are 34.2 million people of all ages with diabetes, according to a report from the CDC in 2018. Diabetes is the leading cause of CKD and ESKD within the United States. The prevalence of CKD is estimated to be 30 million, according to the National Health and Nutrition Examination Survey (NHANES). Within recent years, sodium-glucose co-transporter 2 (SGLT2) inhibitors have gained indications for use in patients with CKD, with or without Diabetes (depending on the agent) and have been shown to prevent progression to ESKD. These agents are also used in the management of blood sugar for T2D. When compared to the SGLT2 inhibitors, Kerendia did not perform as well and also has the limitation of no use for blood sugar control. The estimated cost of Kerendia is $569 per 30-day supply or about $7,000 annually. Peak sales by 2026 are estimated to be close to $533 million. Kerendia may be more useful after a trial and failure on an SGLT2 inhibitor for this patient population.
Saphnelo (anifrolumab), by AstraZeneca/MedImmune
Approved July 2021 for the treatment of adult patients with moderate-to-severe systemic lupus erythematosus (SLE), who are receiving standard therapy. SLE is a chronic, occasionally life-threatening immune disorder that affects multiple organs and can be characterized by the number of antinuclear antibodies (ANA). Management of SLE typically includes treatment with hydroxychloroquine or chloroquine, unless contraindicated. Approximately 80% of patients with non-organ-threatening SLE achieve disease remission with use of these agents. Benlysta (belimumab) is a monoclonal antibody that can be used in conjunction with standard therapy for patients at least 5 years of age for the treatment of SLE. Saphnelo is also a monoclonal antibody that can be used in adult patients with SLE with an estimated cost of $4,600 per vial and close to $60,000 annually when administered every 4 weeks. It is estimated peak sales for Saphnelo will grow to $38 million by 2026. As of December 2020, Benlysta also gained approval for use in lupus nephritis (LN). Based on the ability for Benlysta to be used across SLE and LN, Saphnelo will likely be used after Benlysta failure.
Bylvay (odevixibat), by Albireo
Approved July 2021 for the treatment of pruritus (also known as itching) in patients 3 months of age and older with progressive familial intrahepatic cholestasis (PFIC), which is the defective secretion of bile acids or other components of bile. PFIC is associated with growth failure and progressive liver disease. Pruritus is a primary clinical manifestation in the early stages of PFIC I and II. Conventional therapies (such as ursodiol, antihistamines, or rifampicin) are often unsuccessful in patients with PFIC I and II. Surgical procedures are effective because they interrupt the enterohepatic circulation of bile acids. Bylvay is the first and only approved therapy for PFIC treatment. The annual cost of Bylvay depends on the daily dose used and can range from $80,300 to $963,600. Based on the average weight of the patients in the clinical trials (18kg), the annual cost may be closer to $385,000. Estimated peak sales are predicted to be $580 million by 2026. It works by decreasing the reabsorption of bile acids from the terminal ileum. Bylvay provides a non-surgical option to help manage pruritus in patients with PFIC I and II.
Korsuva (difelikefalin), by Vifor Pharma/Cara Therapeutics
Approved August 2021 for the treatment of moderate-to-severe pruritus associated with chronic kidney disease (CKD-aP) in adults undergoing hemodialysis (HD). According to the Dialysis Outcomes and Practice Patterns Study (DOPPS), the prevalence of pruritus has remained at 18% since the early 2000s. Uremic pruritis is one of the most disabling symptoms for patients with chronic kidney disease. The pruritus is generalized, but most commonly reported on the back with patients experiencing a worsening of symptoms towards the evening hours. Therapy entails optimal dialysis, since underdialysis is more commonly associated with pruritus, and optimal treatment of hyperparathyroidism, hyperphosphatemia, and hypermagnesemia. Emollients, along with other topical analgesics, can also be utilized especially if the patient has dry skin present on examination. Oral antihistamines can also be added onto a patient’s regimen to help alleviate symptoms. Gabapentin was found to be the most effective for patients with uremic pruritus based on a systemic review from 2017. Korsuva, a selective opioid agonist, is the first approved therapy for this indication and was shown to reduce itching in clinical trials when compared to placebo without any opioid-related adverse events. At this moment, cost information is not yet available for Korsuva. Further studies are needed to understand how Korsuva would compare to gabapentin or pregabalin in this patient population.
Welireg (belzutifan), by Merck & Co
Approved August 2021 for the treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery. Von Hippel-Lindau (VHL) is an inherited, autosomal dominant disease that is responsible for both benign and malignant tumors. The prevalence of the VHL gene for VHL disease is about 1 in every 36,000 people and has a mean age of presentation of 26 years. VHL-associated tumors are found in a variety of areas. Management is generally surveillance, unless the patient wants a more aggressive treatment, is symptomatic, or is rapidly progressing. The estimated annual cost is $321,000 per year. Peak sales for 2022 are estimated at $62 million with some suggesting upwards of $274 to $386 million by 2026. Welireg can be used in these situations and can provide an alternative to surveillance and may potentially postpone surgical intervention.
Qulipta (atogepant), by Allergan/AbbVie
Approved September 2021 for the preventive treatment of episodic migraine in adults. Migraines are a neurological disease that, for some people, can be incapacitating. Migraines can affect overall workplace productivity and quality of life. Calcitonin-gene related peptide (CGRP) antagonists are a relatively new class of agents that have gained approval in various formulations for the treatment of acute and prevention of episodic and chronic migraines. Qulipta, an oral CGRP, performed similarly to other CGRPs in the class with a reduction of monthly migraine days of 1 to 2 days when compared to placebo. The annual cost of Qulipta equates to $12,100 per year, at $33.03 per tablet. Estimated peak sales range from $1 billion to $1.2 billion by 2023, but some are suggesting the $1 billion by 2025. The American Headache Society (AHS) released a statement in 2018 that advised CGRPs should be used after traditional therapies for migraines due to significant cost difference.
Livmarli (maralixibat), by Shire/Takeda/Mirum
Approved September 2021 for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older. Cholestasis is an impairment in the excretion of the bile or decreased bile formation, which leads to elevated bilirubin levels within the body. Neonatal cholestasis refers to when this occurs at birth or develops within the first few months of life. Pruritus, a common clinical manifestation of cholestasis, is often treated with ursodeoxycholic acid, rifampin, or bile acid sequestrants (such as cholestyramine, colesevelam) with varying degrees of success. Naltrexone can also be used in the treatment of pruritis in patients with AGLS. Livmarli (maralixibat) is now another option available to patients and has shown some benefit for pruritus in this population. Livmarli therapy, at $1,550 per unit, is estimated to cost $391,000 annually based on the per unit cost of 17kg body weight. Peak sales are estimated at $400 million by 2030. However, in about 40% of AGLS patients, the pruritus becomes refractory to medical treatment and biliary diversion, or liver transplantation may be needed. The goal would be to allow the patient time to outgrow the cholestasis or utilize medical therapy to postpone the need for surgical intervention, as tolerated.
Organizations should carefully consider the clinical efficacy and safety of these new drugs as well as cost and how these latest advancements may impact your formularies, members and patients. If you have specific questions on how new drugs to market may impact your organization, please contact Emily Reonegro at firstname.lastname@example.org.
RemedyOne is a formulary optimization company, focused on leveraging the experience of its healthcare professionals to improve outcomes for patients, employers, providers, drug companies, insurers, pharmacies, and more. Our team’s deep expertise, unique relationships combined with a depth understanding of the complex systems helps us cut through the complexity of modern pharmaceutical programs to uncover opportunities for better outcomes that more people can benefit from.