Regulatory Updates

Niraparib and abiraterone acetate plus prednisone has potential to address unmet need for patients with BRCA-positive metastatic castration-resistant prostate cancer.

If approved, ADX-2191 could be the first marketed drug specifically for patients with primary vitreoretinal lymphoma. The FDA assigned a Prescription Drug User Fee Act date of June 21, 2023.

Kevzara is the first biologic to treat patients with polymyalgia rheumatica (PMR). It is also approved to treat rheumatoid arthritis.

If authorized, Pfizer’s RSV vaccine would be used to prevent respiratory disease caused by RSV in adults 60 years of age and older. An FDA decision is expected by May 2023.

The companies have also submitted an EUA for a fourth dose of the bivalent COVID-19 vaccine in children 6 months through 4 years of age.

Skyclarys will be available in the second quarter of 2023 to treat patients with Friedreich’s ataxia, an ultra-rare, inherited neurodegenerative disorder. The wholesale acquisition cost is $370,000.

BMS is seeking an indication for Opdivo as monotherapy to treat patients with stage IIB or IIC melanoma after surgery. The FDA has assigned a Prescription Drug User Fee Act date of Oct. 13, 2023.

Altuviiio is a first-in-class therapy providing factor VIII replacement to prevent bleeding in patients with hemophilia A. It is expected to be available in April 2023.

If approved, pozelimab would be the first treatment for CHAPLE disease, which causes damage to blood and lymph vessels along the upper digestive tract. The target action date for the FDA decision is Aug. 20, 2023.

CSF-1 is a low-dose pilocarpine to treat patients with age-related blurry vision. The FDA has assigned a PDUFA goal date of Oct. 22, 2023

Alnylam is seeking approval for Onpattro’s use in cardiomyopathy related to transthyretin-mediated (ATTR) amyloidosis. The FDA set an action date of Oct. 8, 2023.

The new once-daily formulation of Austedo is used to treat adults with tardive dyskinesia and chorea associated with Huntington’s disease.

If approved, RSVpreF would be the first vaccine for administration to pregnant women to help protect against RSV disease in infants. The FDA has set an action date for August 2023.

Filspari will be available for a wholesale acquisition cost of $9,900 for a 30-day supply, and administrated through a REMS program because of the risk of liver abnormalities.

Apellis’ Syfovre will have a list price of $2,190 per vial, and Medicare is expected to be the dominant payer.

The review has been extended by three months. The new Prescription Drug User Fee Act (PDUFA) date is May 22, 2023.

Lamzeda has been approved to treat patients with alpha-mannosidosis, a rare genetic condition. It will be available in the first half of this year.

The Prescription Drug User Fee Act action date is June 5, 2023. Prevymis is already approved to prevent CMV infection in adults after an allogeneic hematopoietic stem cell transplant.

Avacincaptad pegol has the potential to slow the progression of geographic atrophy in age-related macular degeneration. The PDUFA goal date is Aug. 19, 2023.

Phase 3 data showed that the combination resulted in a 37% reduction in risk of disease progression or death in men with advanced prostate cancer.

The FDA indicated that Soligenix’s application for HyBryte, a novel photodynamic therapy, was incomplete.

If approved, Linzess would be the first and only FDA-approved prescription therapy for functional constipation in children 6 to 17 years of age. The PDUFA date is June 14th, 2023.

Cibinqo is an oral, once-daily JAK1 inhibitor now approved for those 12 years of age and older.

High-priced gene therapies challenge payers to provide access. They are watching the products to be approved the first half of this year.

Conversion from accelerated to regular approval was based on long-term outcomes from a phase 1 trial, which demonstrated an overall response rate of 45.4%.

Eylea now also treats retinopathy of prematurity, a leading cause of childhood blindness worldwide.

If approved, reproxalap would be the first inhibitor of RASP to treat patients with dry eye disease. The FDA PDUFA date is Nov. 23, 2023.

Takhzyro is the first therapy to prevent attacks of hereditary angioedema (HAE) in children ages 2 to under 6.

Zuranolone is a rapid-acting neuroactive steroid that can take effect in 14 days to treat patients with major depressive disorder and postpartum depression. The FDA has assigned a PDUFA action date of Aug. 5, 2023.

The products may be ineffective, unsafe and could prevent a person from seeking an appropriate diagnosis.