Regulatory Updates

In a complete response letter, the FDA has requested information about the pump that will be used to deliver ABBV-951, which will provide continuous subcutaneous delivery of oral immediate-release carbidopa/levodopa.

Evkeeza treats a rare form of high cholesterol. It is now approved for patients as young of 5 years of age.

Illuccix is for positron emission tomography (PET) imaging of prostate-specific membrane antigen (PSMA) positive lesions in men with prostate cancer.

Daybue will be available by the end of April with a list price of about $375,000 annually.

Zavzpret is the first calcitonin gene-related peptide (CGRP) receptor antagonist nasal spray approved for patients with acute migraine.

Other news includes the launches of the first generic of an ulcer drug, the antipsychotic lurasidone, an extended-release version of topiramate for migraine, and additional strengths of the cancer drug lenalidomide. Additionally, Glenmark will distribute Cediprof's generic Adderall, and the FDA has accepted Amneal’s application for a generic naloxone nasal spray.

If approved, Jardiance would be the first SGLT2 inhibitor indicated for this population. A decision is expected in the second quarter of 2023.

Voxzogo is already available to treat patients older than five years with a genetic cause of dwarfism. The FDA has set a PDUFA of Oct. 21, 2023, for children younger than 5.

If approved, berdazimer could be the first prescription product treat molluscum, a common skin infection caused by poxvirus. The Prescription Drug User Fee Act goal date is Jan. 5, 2024.

With a Prescription Drug User Fee Act action date of Dec. 22, 2023, eplontersen is an antisense medicine to treat patients living with hereditary transthyretin-mediated amyloid polyneuropathy, which results in nerve damage.

The agency has set a new PDUFA target action date of June 30, 2023, for Roctavian. If approved, it would be the first one-time gene therapy for hemophilia. A.

The FDA plans to hold an advisory committee meeting to discuss the Alzheimer’s therapy. The agency has assigned Prescription Drug User Fee Act target action of July 6, 2023.

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.