Regulatory Updates

Brexafemme is a non-azole oral antifungal now approved to both treat and prevent recurrent yeast infections in women.

I/Ontak is an engineered IL-2-diphtheria toxin fusion protein. The PDUFA target action date is Sept. 28, 2023.

Rebyota is the first fecal microbiota product to prevent recurrence of C. diff infection, a serious and potentially life-threatening disease.

If approved, Evkeeza would be the first treatment to help children as young as 5 years old control high LDL cholesterol caused by homozygous familial hypercholesterolemia. The FDA target action date is March 30, 2023.

A confirmatory trial of Tecentriq in urothelial carcinoma failed to meet the co-primary endpoint of overall survival.

If approved, reproxalap would be the first inhibitor of RASP, which contributes to ocular inflammation and changes in tear lipid composition.

If approved, SRP-9001 (delandistrogene moxeparvovec) would be the first gene therapy for Duchenne. The FDA action date is May 29, 2023.

An advisory committee had voted against poziotinib to treat patients with non-small cell lung cancer with HER2 exon 20 insertion mutations.

An FDA decision on Takeda’s dengue vaccine TAK-003 is expected within six months.

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.

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.

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.