Politics and Policy

Cell and gene therapies can be used for patients with cancer and serious diseases who have limited or no treatment options — but at a cost.

In clinical trials, Aphexda plus filgrastim enabled a majority of patients to achieve the collection goal to enable stem cell transplantation for patients with multiple myeloma. It will have a list price 5,900 per vial, with most patients needing two vials for treatment.

Libervant is film version of diazepam that is placed inside the cheek to treat children between two and five years of age who have refractory epilepsy. The PDUFA target goal date is April 28, 2024.

If approved, mavorixafor would be the first therapy to address the genetic defect that results in WHIM syndrome, an ultra-rare disease that can cause recurrent lung infections, papillomavirus-related warts, and an increased risk of developing certain types of cancer.

With this tentative approval of abacavir/dolutegravir/lamivudine, Viatris aims to help adherence to HIV treatment in low- and middle-income countries.

The anti-cancer drug bevacizumab is used off-label to treat wet AMD. Outlook Therapeutics had developed an ophthalmic formulation of bevacizumab for intravitreal injection.

At least 15 manufacturers have received approval for both capsule and chewable tablet generic versions of Vyvanse.

In head-to-head study, Reblozyl nearly doubled the percent of patients achieving primary endpoint of concurrent transfusion independence and hemoglobin increase vs. epoetin alfa.

The FDA granted accelerated approval to Balversa in 2019. Patients who received Balversa in Janssen’s confirmatory study achieved a median overall survival of more than one year. It reduced the risk of death by 36%.

The submissions to both the FDA and the EMA are supported by two phase 3 trials demonstrating Skyrizi achieved the primary endpoint of clinical remission. Skyrizi is already available to treat patients with plaque psoriasis, psoriatic arthritis, and Crohn’s disease.

Sandoz’s Tyruko, the first biosimilar to Biogen’s Tysabri, is expected to be available as soon as possible. Pricing has not yet been made available.

If approved, Xtandi would be the first hormonal therapy for non-metastatic patients with increasing PSA levels. The FDA has assigned an action date in the quarter of 2023.

The FDA set an action date of June 16, 2024, for imetelstat to treat transfusion-dependent anemia in myelodysplastic syndromes. Regulators said they plan to hold an advisory committee meeting as part of their review.

Abrysvo is the first RSV vaccine approved to be given to pregnant women at 32 to 36 weeks to prevent infection in infants.

In addition to tardive dyskinesia, Ingrezza now also treats chorea associated with Huntington’s disease, which is a movement disorder that effects coordination, gait, swallowing and speech.

The higher dose of Eylea allows longer intervals between injections for patients with macular degeneration and diabetic retinopathy. It will have a list price of $2,625 per single-use vial.

Veopoz is the first treatment for CHAPLE disease, which causes damage to blood and lymph vessels along the upper digestive tract. The list price is $34,615.38 per single-use vial.

The FDA granted accelerated approval to Tarpeyo in December 2021 to treat patients with primary IgA nephropathy, a rare disease that attacks the kidneys. The action date for full approval is Dec. 20, 2023.

Sohonos was approved to treat children with fibrodysplasia ossificans progressive, an ultra-rare disease that transforms the body’s soft tissue into bone. It has an annual list price of $624,000.

Tibsovo is currently approved in the United States as a monotherapy to treat adults with IDH1-mutant relapsed or refractory acute myeloid leukemia.

The FDA has granted priority review for cefepime-taniborbactam to treat patients with complicated urinary tract infections and set an action date of Feb. 22, 2024.

Elrexfio is a bispecific antibody to treat relapsed or refractory multiple myeloma. It will be available within the next few weeks and have a monthly list price of $41,500.

Daxxify is a long-acting botulinum toxin that will launch in 2024. It previously received FDA approval to treat frown lines.

The combination of niraparib and abiraterone in a single tablet — now with the name Akeega — will be available in mid-September and have a list price of $18,750 for a 30-day supply.

Talvey is a first-in-class bispecific antibody to treat adult patients with relapsed or refractory multiple myeloma. It will available in a few weeks and have a price of $45,000 per month.

Avasopasem was under review to treat radiotherapy-induced severe oral mucositis in patients with head and neck cancer. The FDA has indicted that an additional trial would be needed to show safety and effectiveness of avasopasem.

The FDA has assigned a March 8, 2024, action date for GA Depot, a once-monthly injection to treat patients with relapsing forms of multiple sclerosis.

Izervay, which is expected to be available within the two to four weeks, will have a list price of $2,100 per vial.

Zurzuvae is a rapid-acting neuroactive steroid that is expected to be available in the fourth quarter of 2023.

Trial results showed that the combination of Lonsurf and bevacizumab provided improvements in overall survival.