Politics and Policy

Hemgenix is the first one-time gene therapy treatment for adults with hemophilia B. It will have a list price of $3.5 million.

An FDA review has found that Prolia can result in low calcium levels in the blood in patients with advanced kidney disease and can lead to hospitalization and death.

A confirmatory trial in patients with relapsed or refractory multiple myeloma did not meet its primary endpoint of progression-free survival.

If approved, epcoritamab would be the first bispecific antibody to treat adult patients with relapsed/refractory large B-cell lymphoma. The Prescription Drug User Fee Act date is May 21, 2023.

The reference to “maximum fair price” in the act bodes poorly for manufacturers and suggests more of a take-it-or-leave-it situation rather than a negotiation where clinical evidence would be the prevailing factor in determining price.

Appellis had submitted updated data for the pegcetacoplan in age-related macular degeneration, which is considered a major amendment to the NDA. The new PDUFA date is Feb. 26, 2023.

Rylaze’s dosing options provide sustained asparaginase activity throughout the entire course of treatment for adult and pediatric patients with acute lymphoblastic leukemia or lymphoblastic lymphoma.

Lilly’s Rezvoglar is a long-acting human insulin analog. It follows Semglee as a biosimilar that can be substituted for Lantus for both adults and children with diabetes.

Tzield addresses the underlying cause of the disease, where the immune system destroys the insulin producing cells in the pancreas. It will be available by the end of the year with a wholesale acquisition cost of $13,850 per vial.

If approved, Xphozah will be the first phosphate absorption inhibitor for adults with chronic kidney disease who are on dialysis. An FDA decision is expected within 30 days.

The agency is encouraging sponsors to submit applications for low-dose, nonprescription naloxone products.

After review of phase 3 data, GSK will limit the use of Zejula in the second line to those with inherited BRCA mutations.

Elahere is a first-in-class antibody-drug conjugate targeted against folate receptor alpha, a protein on the surface of ovarian cancer cells.

The approval extends the use of Liletta for an additional two years. It is the only hormonal IUD approved for continuous use up to eight years.

Zilucoplan is a targeted therapy that inhibits key components in the underlying disease pathology. The anticipated PDUFA date is in the fourth quarter of 2023.

PRX-102 is an enzyme replacement therapy for Fabry disease, a rare genetic disorder.

Imjudo is a monoclonal antibody that targets the activity CTLA-4 that was approved to be used in combination with Imfinzi, a PD-L1 inhibitor, for patients with non-small cell lung cancer.

IPX203 is an oral formulation of carbidopa/levodopa extended-release capsules that, if approved, could provide patients with better symptom control. The FDA assigned a Prescription Drug User Fee Act date of June 30, 2023.

In a trial of patients two years and older, Adcetris plus standard of care chemotherapy led to a 59% reduction in risk of disease progression or relapse, second malignancy or death

If approved, Aphexdra would be used along with G-CSF treatment for autologous transplantation in patients with multiple myeloma. The Prescription Drug User Fee Act target action date is Sept. 9, 2023.

Kineret has been granted an EUA for hospitalized adults with pneumonia who require supplemental oxygen.

Abortion access was a key issue in Pennsylvania and other battleground states. The Affordable Care Act receded into the political background, although voters in South Dakota approved a ballot measure that expanded Medicaid, leaving just 11 states that haven't done so.

The new indication expands the patient population eligible for a Libtayo-based regimen to include combination treatment with chemotherapy irrespective of PD-L1 expression levels.

Cotellic, an oral inhibitor of MEK1 and MEK2, was approved to treat patients with histiocytic neoplasms based on a phase 2 trial conducted solely at Memorial Sloan Kettering Cancer Center.

This approval expands the use of Vemlidy to children 12 years of age and older. Vemlidy is already available for adults living with this chronic liver disease.

If approved, GSK’s vaccine could be the first available to help protect adults over the age of 60. The Prescription Drug User Fee Act date is May 3, 2023.

The withdrawal is related to the quality of the data collection and monitoring of the pivotal clinical trials by the clinical research organization.

The FDA cited COVID-19-related travel restrictions that have impacted the agency’s inspection of a manufacturing site in China.

If South Dakota voters approve a ballot initiative on Nov. 8, that will leave 11 states that have not expanded their Medicaid programs under the ACA.

Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B-cells