Denise Myshko

Sarepta continues to work with regulators to complete the safety label update for Elevidys, and they are discussing an approach for risk mitigation for non-ambulatory patients.

Two PBMs, True Rx Health Strategies and Capital Rx, are using pharmacogenomics — how a person’s DNA affects their response to medications — to reduce the trial-and-error of prescribing medications, saving employers and patients time and money.

Health policy researcher Geoffrey Joyce argues that only delinking compensation from the list price of a drug will lower drug spending.

Last week, an FDA advisory committee against the risk-benefit profile of Blenrep in combination with other therapies. Regulators and reviewers were concerned about the ocular side effects and dosing and tolerability. The new action date is Oct. 23, 2025.

The Vision Center at Children’s Hospital Los Angeles is developing a protocol for a phase 1 clinical trial for the first gene therapy for boys with blue cone monochromacy, which impairs color vision and severely affects visual function.

Sarepta officials said the temporary halt in shipments was done to maintain a productive working relationship with regulators while they address a safety labeling update about the risk of acute liver disease related to Elevidys.

Icotrokinra is a first-in-class targeted oral peptide that blocks interleukin (IL) 23 and its receptor, which plays a key role in the inflammatory response in plaque psoriasis.

Elevidys is a gene therapy approved to treat Duchenne muscular dystrophy. Last month, Sarapta halted sales of the therapy for non-ambulatory patients.

In early research, OCU410ST, a gene therapy for Stargardt disease, demonstrated slowed lesion growth by 48% at the 12-month follow-up in evaluable treated eyes.

The combination of RPI, a genetically engineered herpes simplex virus type 1, and Opdivo shrank both injected and non-injected tumors by 30%.

Following in the footsteps of other pharmaceutical companies, Bristol Myers Squibb and Pfizer are offering Eliquis for cash-paying patients at a discount of 40% off the list price.

A class of drugs, MDM2 inhibitors, being studied as cancer therapeutics may accidentally damage the protective barriers in the brain and eyes, finds new research in mice.

Despite the recent approval of lenacapavir as a twice yearly PrEP, there is still a need for choice in HIV prevention, argue speakers at a session at the International AIDS Society meeting.

Global health leaders and advocates challenged attendees of IAS to support the work of the partnerships that are creating patient-focused innovations for HIV prevention and treatment.

Increasing the use of Apretude among Black women requires not only expanding awareness of the long-acting PrEP regimen in various clinics but also providing more flexible scheduling and partnering with specialty pharmacies.

Barbie wears a continuous glucose monitor and an insulin pump and has a phone displaying a CGM app.

After a one-year follow-up, 10 of 12 patients given the islet cell therapy zimislecel no longer required insulin. Vertex officials said regulatory submissions are expected in 2026

Employers, some lawmakers and others have criticized pharmacy benefit managers (PBMs), especially the "big three," for having complicated, sometimes hidden practices. The PBMs say they have responded with programs and policies that add transparency to what they do.

For payers, claims for rare disease treatments used to be uncommon. Not anymore, thanks to an increasing number of new, high-priced drugs for rare diseases.

Optum Rx officials said they now have eliminated reauthorization requirements for more than 140 chronic disease medications.

A separate Prime analysis, however, of year-over-year claims data for Wegovy and Zepbound only found an improvement in adherence in 2024, suggesting there is an evolving understanding of obesity treatment.

A new study finds that enrollees who had difficulty accessing care in Medicare Advantage plans were more likely to switch to traditional Medicare than to another MA plan.

More than 50 insurers have pledged to streamline and simplify the prior authorization process through six new commitments.

The FDA had resolved the shortage of Wegovy in April, and telehealth providers were advised to stop selling compounded semaglutide products. Novo Nordisk said that Hims & Hers continues to offer these compounded drugs.

Researchers at Yale School of Medicine have determined that a panel of biomarkers can help physicians better understand kidney disease in children.

More than 99% effective at preventing HIV in trials, Yeztugo (lenacapavir) is now the first and only twice-yearly option for pre-exposure prophylaxis (PrEP).

Wyost and Jubbonti are interchangeable with Xgeva/Prolia and are approved for all of the same indications in bone cancer and osteoporosis.

A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys.

In an interview, James D. Chambers, Ph.D., from Tufts Medical Center, talks about the factors that are impacting the biosimilar market.