September 29th 2023
The two-component therapy of Pombiliti plus Opfolda to treat adults living with late-onset Pompe disease will have an annual list price of $650,000.
Odronextamab is a bispecific antibody to treat relapsed/refractory follicular lymphoma and diffuse large B-cell lymphoma. The target action date is March 31, 2024.
September 28th 2023
Exxua is approved to treat adults with major depressive disorder, but its labeling does not contain warnings about sexual function or weight gain.
Sotatercept is a first-in-class therapy to treat the rare disease pulmonary arterial hypertension. The FDA has assigned a target action date of March 26, 2024.
September 27th 2023
Ryzumi is expected to be available in the first half of 2024. Pricing will be available around the time of launch.
Janssen Submits NDA for Combo Tablet for Metastatic Prostate Cancer
Niraparib and abiraterone acetate plus prednisone has potential to address unmet need for patients with BRCA-positive metastatic castration-resistant prostate cancer.
FDA Accepts for Priority Review Drug for Rare Retinal 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.
FDA Approves Kevzara for Inflammatory Rheumatic Disease
Kevzara is the first biologic to treat patients with polymyalgia rheumatica (PMR). It is also approved to treat rheumatoid arthritis.
FDA Advisory Committee Supports Pfizer’s RSV Vaccine
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.
Pfizer, BioNTech Seek Approval for Bivalent COVID-19 Vaccine as Primary Series
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.
FDA Approves First Therapy for Rare Neuromuscular Disease
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.
FDA Sets Action Date for Opdivo for Melanoma Indication
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.
FDA Approves Weekly Hemophilia A Therapy
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.
FDA Grants Priority Review for Therapy for Life-threatening Hereditary Immune Disease
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.
FDA Sets Goal Date for Blurry Vision Therapy
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
FDA to Hold Advisory Committee Meeting on Onpattro for Heart Failure Indication
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.
FDA Approves Extended Release Austedo
The new once-daily formulation of Austedo is used to treat adults with tardive dyskinesia and chorea associated with Huntington’s disease.
FDA Grants Priority Review for Pfizer’s RSV Vaccine
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.
FDA Grants Accelerated Approval to Filspari for Rare Kidney Disease
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.
FDA Approves First Drug for Geographic Atrophy Due to AMD
Apellis’ Syfovre will have a list price of $2,190 per vial, and Medicare is expected to be the dominant payer.
FDA Extends Review of Fezolinetant for Menopause Symptoms
The review has been extended by three months. The new Prescription Drug User Fee Act (PDUFA) date is May 22, 2023.
Approves Enzyme Replacement Therapy for Rare Disease
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.
FDA Grants Priority Review to Prevymis to Prevent CMV After Kidney Transplants
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.
FDA Grants Priority Review to AMD Therapy
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.
FDA Grants Priority Review Talzenna/Xtandi Combo in Prostate Cancer
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.
FDA Refuses NDA for Cutaneous T-Cell Lymphoma
The FDA indicated that Soligenix’s application for HyBryte, a novel photodynamic therapy, was incomplete.
FDA Grants Priority Review for Linzess in Children, Adolescents
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.
FDA Expands Cibinqo Label for Atopic Dermatitis in Adolescents
Cibinqo is an oral, once-daily JAK1 inhibitor now approved for those 12 years of age and older.
3 New, and High-Cost, Gene Therapies are Expected This Year
High-priced gene therapies challenge payers to provide access. They are watching the products to be approved the first half of this year.
FDA Grants Full Approval to Jemperli for Endometrial Cancer
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%.
FDA Approves First Pediatric Indication for Blockbuster Eylea
Eylea now also treats retinopathy of prematurity, a leading cause of childhood blindness worldwide.
FDA Sets PDUFA Date for Dry Eye Disease Drug
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.
FDA Okays Pediatric Indication for Takeda’s Takhzyro
Takhzyro is the first therapy to prevent attacks of hereditary angioedema (HAE) in children ages 2 to under 6.
FDA Accepts NDA for Zuranolone for Depression
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.
FDA Warns Companies Claiming to Treat Monkeypox
The products may be ineffective, unsafe and could prevent a person from seeking an appropriate diagnosis.
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