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.
FDA Approves Second Indication for Brexafemme
Brexafemme is a non-azole oral antifungal now approved to both treat and prevent recurrent yeast infections in women.
FDA Accepts BLA for Cutaneous T-Cell Lymphoma Therapy
I/Ontak is an engineered IL-2-diphtheria toxin fusion protein. The PDUFA target action date is Sept. 28, 2023.
FDA Approves Rebyota, the First-ever Microbiota Product
Rebyota is the first fecal microbiota product to prevent recurrence of C. diff infection, a serious and potentially life-threatening disease.
FDA Accepts sBLA for Evkeeza for Children with Rare High Cholesterol
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.
Genentech Withdraws Tecentriq Indication for Bladder Cancer
A confirmatory trial of Tecentriq in urothelial carcinoma failed to meet the co-primary endpoint of overall survival.
Aldeyra Submits NDA for the Novel Dry Eye Therapy Reproxalap
If approved, reproxalap would be the first inhibitor of RASP, which contributes to ocular inflammation and changes in tear lipid composition.
FDA Grants Priority Review for Duchenne Muscular Dystrophy Gene Therapy
If approved, SRP-9001 (delandistrogene moxeparvovec) would be the first gene therapy for Duchenne. The FDA action date is May 29, 2023.
FDA Issues CRL for Poziotinib for Lung Cancer
An advisory committee had voted against poziotinib to treat patients with non-small cell lung cancer with HER2 exon 20 insertion mutations.
FDA Grants Priority Review of BLA for Dengue Vaccine
An FDA decision on Takeda’s dengue vaccine TAK-003 is expected within six months.
Updated: FDA Approves $3.5 million Gene Therapy Hemgenix for Hemophilia B
Hemgenix is the first one-time gene therapy treatment for adults with hemophilia B. It will have a list price of $3.5 million.
FDA Warns About Prolia in Patients with Kidney Disease
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.
GSK Pulls Blenrep from U.S. Market
A confirmatory trial in patients with relapsed or refractory multiple myeloma did not meet its primary endpoint of progression-free survival.
FDA Accepts for Priority Review BLA for Epcoritamab
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.
FDA Updates Target Date for Pegcetacoplan
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.
FDA Approves Alternate Dosing Schedule for Rylaze
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.
FDA Approves Second Interchangeable Lantus Biosimilar
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.
FDA Approves First Drug that Delays 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.
FDA Advisory Committee Supports Xphozah in CKD
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.
FDA Proposes Prescription-to-OTC Naloxone
The agency is encouraging sponsors to submit applications for low-dose, nonprescription naloxone products.
GSK Limits Use of Zejula for Some Cancer Patients
After review of phase 3 data, GSK will limit the use of Zejula in the second line to those with inherited BRCA mutations.
FDA Grants Accelerated Approval of Novel Ovarian Cancer Therapy
Elahere is a first-in-class antibody-drug conjugate targeted against folate receptor alpha, a protein on the surface of ovarian cancer cells.
FDA Approves Liletta IUD to Prevent Pregnancy for 8 Years
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.
FDA Accepts NDA for Generalized Myasthenia Gravis Therapy
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.
Protalix, Chiesi Resubmit BLA to Treat Fabry Disease
PRX-102 is an enzyme replacement therapy for Fabry disease, a rare genetic disorder.
FDA Clears Imfinzi/Imjudo Combination for Lung Cancer
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.
FDA Accepts NDA for Novel Parkinson’s Disease Therapy
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.
FDA Approves Adcetris for Younger Patients with Hodgkin Lymphoma
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
FDA Accepts NDA of Aphexda for Use in Stem Cell Mobilization
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.
FDA Issues EUA for Kineret for Hospitalized COVID-19 Patients
Kineret has been granted an EUA for hospitalized adults with pneumonia who require supplemental oxygen.
FDA Approves Libtayo for Second Indication in Advanced Lung Cancer
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.
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