Regulatory Updates

Exagamglogene autotemcel is being evaluated for patients with sickle cell disease and beta thalassemia, in which a patient’s own hematopoietic stem cells are edited to produce high levels of fetal hemoglobin in red blood cells.

Vevye (previously CyclASol) is a water-free, preservative-free solution, which allows for improved bioavailability and better efficacy on the target tissue.

The FDA has assigned an action date of Sept. 23, 2023. If approved, this would be the first frontline treatment advancement in decades for patients with advanced or recurrent endometrial cancer.

Prevymis was approved to prevent CMV infection in adults after an allogeneic hematopoietic stem cell transplant in 2017.

The combination of Lynparza and abiraterone reduced the risk of disease progression or death by 76% vs. abiraterone alone in patients with BRCA-mutated metastatic castration-resistant prostate cancer.

Miebo is the first and only prescription eye drop approved for dry eye disease (DED) that directly targets tear evaporation.

Lifileucel is a polyclonal tumor infiltrating lymphocyte (TIL) therapy designed for patients with advanced melanoma who have experienced progression after previous treatment with anti-PD-1/L1 therapy and targeted therapy.

The frequency of drug manufacturer coupon utilization is associated with more market competition—but not patients’ out-of-pocket costs.

More than 11.6 million treatment courses of the medication have been prescribed in the U.S. to date.

The FDA recently approved Brixadi to treat moderate to severe opioid use disorder in patients who have already started treatment with a transmucosal form of buprenorphine, and have approved Opvee as an emergency treatment to reverse known or suspected opioid overdose in people ages 12 years and older.

The list price for a 30-day supply of the drug, Rinvoq, is around $6,125. Eligible patients may be able to benefit from copay savings cards and other financial support programs from AbbVie.

The compulsory orders will require the GPOs, which negotiate drug rebates on behalf of other PBMs, to provide information and records on their business practices.

A wholesale acquisition cost (WAC) for Elfabrio will be announced once it is available in a few weeks.

Using a national database, data on medication purchases was collected, along with factors that may have influenced expenditures, such as new therapy availability, patent expirations, policy changes, and legislation.

Cancer-related cost-effective analyses (CEA) referenced higher thresholds than non-cancer-related CEAs, which may suggest interventions for diseases associated with greater mortality and morbidity warrant higher thresholds.

In June, the Advisory Committee on Immunization Practices (ACIP) will make recommendations on the appropriate use of the vaccine in the U.S.

Cystic fibrosis drug Trikafta gets expanded indication for children ages 2-5.

Uzedy’s wholesale acquisition cost (WAC) ranges from $1,232 to $3,080 per month.

CDI is a leading cause of infections acquired in healthcare settings, including hospitals and nursing homes. After recovering, individuals may get the infection again, and the risk of recurrence increases with each infection.

The list price for Qalsody, the first and only approved treatment to target a genetic cause of ALS, may prove to be controversial.

The expansion offers increased reimbursement opportunities and additional care services to rural independent pharmacies. In addition, it creates an Independent Pharmacy Advisory Committee to expand the role of rural, suburban and urban pharmacies in the healthcare system.

Omisirge is a stem cell therapy that helps the body recover more quickly from cancer treatments that kill normal blood cells along with blood cancer cells, thus lowering the risk of infection.

The FDA cited issues related to the proposed manufacturing of the drug, mirikizumab. Lilly expressed confidence that they could be corrected.

Through Express Scripts’ new Copay Assurance plan, consumers will have lower out-of-pocket costs because of caps on prescription drugs: $5 for generics, $25 for preferred brand drugs, and $45 for preferred specialty brand drugs.

Emergent BioSolutions hasn’t provided a price for the nonprescription Narcan but said the product will be available by late summer.

If approved, Perrigo’s Opill could be the first-ever nonprescription birth control pill.

Leniolisib, now with the brand name Joenja, treats APDA, a genetic disorder that impairs the immune system. It will have an annual list price of $547,500 a year.

Rezzayo is a novel once-weekly antifungal approved to treat invasive candidiasis, a serious infection that can affect the blood, heart and brain.

Regulatory officials identified additional requirements for approval of an extended-release version of Jakafi, which is under review for myelofibrosis, polycythemia vera and graft-versus-host disease.

Zynyx will be priced comparable to other PD-1 inhibitors currently available to treat metastatic or recurrent locally advanced Merkel cell carcinoma, a rare skin cancer.