SPOTLIGHT -
All
FDA Decision on Donanemab for Alzheimer’s Expected by End of Year
Donanemab slowed cognitive decline by about 35% for patients at the earliest stages of Alzheimer’s disease.
FDA Approves First Nonprescription Oral Contraceptive
Opill will be available in stores and online in the first quarter of 2024.
Takeda Pulls Application for Dengue Vaccine
Takeda indicated that it could not address issues related to aspects of data collection within the current BLA review cycle.
FDA Approves Leqvio for Earlier Use in Heart Disease
The label for Leqvio has also been updated to remove four adverse events based on recent safety data.
CMS Expands Leqembi Coverage After FDA’s Full Approval
Medicare will cover Alzheimer’s drugs with full approval, including Leqembi — if the physician and patient participate in a real-world registry trial to gather additional data.
FDA Sets Action Date for Zolbetuximab for Stomach Cancers
Zolbetuximab is a first-in-class therapy for advanced gastric and gastroesophageal cancers. The target action date is Jan. 12, 2024.
Prime Therapeutics Names Mostafa Kamal President and CEO
The Magellan Rx Management CEO is replacing Ken Paulus, who has held the top job at Prime since May 2019.
FDA Rejects Amneal’s Application for Novel Parkinson’s Therapy
The FDA is asking for more pharmacokinetic data on carbidopa, one of the drugs within IPX203. The therapy is an extended-release, oral formulation of carbidopa/levodopa.
FDA Approves $2.9 Million Gene Therapy for Hemophilia A
BioMarin is offering an outcomes-based warranty that will reimburse payers up to 100% of the cost if a patient does not respond to Roctavian.
New Pediatric Growth Hormone Therapy Costs $8,300 Monthly
Longer-acting growth hormone reduces frequency of injections. It will be available in August.
FDA Approves First Cellular Therapy for Type 1 Diabetes
In two small studies, patients were able to be free from insulin injections for a year or more.
FDA Rejects Higher Dose of Eylea
At issue is an ongoing review of inspection results from a third-party filling company. No issues were cited in the FDA’s complete response letter about clinical efficacy or safety.
FDA Approves Rystiggo for Generalized Myasthenia Gravis
It will be commercially available in the third quarter of 2023 and have a list price of $6,050 per vial.
FDA Sets Action Date for Hemophilia B Gene Therapy
Pfizer’s biologics license application (BLA) for the gene therapy fidanacogene elaparvovec has been assigned a PDUFA date in the second quarter of 2024.
FDA Converts Blincyto to Full Approval for Minimal Residual Disease in B-Cell ALL
Blincyto is immuno-oncology therapy that targets CD19 surface antigens on B cells to treat patients with acute lymphoblastic leukemia. Its current wholesale acquisition cost price is $4,900.15 per vial.
FDA Approves Pfizer’s Alopecia Drug
Now with the name Litfulo, ritlecitinib is the first oral treatment for adolescents with alopecia. It has an annual list price of $49,000.
First Gene Therapy for Duchenne Priced at $3.2 Million
Elevidys is a one-time therapy that delivers to muscle a gene that codes for a shortened, functional form of dystrophin, which is mutated in Duchenne muscular dystrophy.
FDA Rejects NDA for Fatty Liver Disease Therapy
The company plans to discontinue further research of obeticholic acid to treat nonalcoholic steatohepatitis (NASH) after the FDA asked for long-term outcomes data.
FDA Issues CRL for Treatment for Rare Retinal Cancer
Regulators indicated there was a lack of evidence of effectiveness and lack of clinical trials done to support the application.
FDA Approves Subcutaneous Vyvgart for Myasthenia Gravis
Vyvgart Hytrulo’s under-the-skin administration means it can be given in 30 to 90 seconds, compared with one hour for the intravenous product. It will be priced at parity to Vyvgart, which has a net price of $225,000 annually.
FDA Clears Jardiance and Synjardy for Kids with Type 2 Diabetes
Jardiance and Synjardy are the first SGLT2 inhibitors approved for children with type 2 diabetes.
Geron Submits NDA for First-in-Class Therapy for MDS
Imetelstat targets telomerase to inhibit uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies.
FDA Approves First Anti-inflammatory Drug for Cardiovascular Disease
Lodoco targets the inflammation that is an underlying cause of atherosclerotic cardiovascular disease. It will be available in the second half of 2023.
FDA Issues Complete Response for Drug for Rare Fungal Infections
The FDA is requesting additional data and analysis for olorofim. F2G plans to resubmit revised NDA with complete data set from phase 2 study.
FDA Approves Second Bispecific Antibody for Diffuse Large B-cell Lymphoma
Columvi will cost about $350,000 for a fixed duration therapy with 12 treatments over eight and a half months.
New Senate Bill Focuses on PBM Compensation in Medicare Plans
If passed, PBMs would not be able to charge fees that are connected to the price of a drug, discounts, or rebates.
FDA Approves Linzess to Treat Children with Constipation
Linzess is first FDA-approved prescription therapy for functional constipation in children 6 to 17 years of age.
FDA Committee Recommends Sanofi/AstraZeneca’s RSV Vaccine for Infants
If approved, nirsevimab would be the first immunization specifically to protect infants through their first RSV season. The Prescription Drug User Fee Act date is in the third quarter of 2023.
FDA Sets PDUFA Dates for First CRISPR Gene Edited Therapy
Exagamglogene autotemcel is being evaluated for patients with sickle cell disease and beta thalassemia, in which a patient’s own hematopoietic stem cells are edited to produce high levels of fetal hemoglobin in red blood cells.
FDA Approves Novel Cyclosporine for Dry Eye Disease
Vevye (previously CyclASol) is a water-free, preservative-free solution, which allows for improved bioavailability and better efficacy on the target tissue.
