Pipeline News and Updates
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Regulatory Update
The FDA approved sulbactam and durlobactam (Xacduro, Innoviva), on 5/23/2023, for the treatment of hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia (HABP/VABP) caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex (Acinetobacter). The FDA approved sotagliflozin (Inpefa, Lexicon Pharmaceuticals), on 5/26/2023, to reduce the risk of cardiovascular death, hospitalization for heart failure, and urgent heart failure in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors. The FDA approved Pfizer’s respiratory syncytial virus vaccine (Abrysvo), on 5/31/2023, to prevent lower respiratory tract infection caused by RSV in individuals 60 and older. The FDA is delaying the PDUFA date for delandistrogene moxeparvovec by one month in order to consider an accelerated approval for Duchenne patients ages 4-5 years old. The new PDUFA date is 6/22/2023. Full approval and expansion to other age groups or withdrawal from the market will depend on the results of the Phase III EMBARK trial. The EMBARK trial is forecast to report top-line results by the end of the year. The FDA accepted the NDA for fruquintinib for the treatment of refractory metastatic colorectal cancer and set a PDUFA date for 11/30/2023. The FDA accepted the BLA for lifileucel for the treatment of unresectable or metastatic melanoma and set a PDUFA date for 11/25/2023 Regulatory Update
The FDA approved beremagene geperpavec (Vyjuvek, Krystal Biotech), on 5/19/2023, for the treatment of dystrophic epidermolysis bullosa. The FDA approved epcoritamab (Epkinly, Genmab & AbbVie), on 5/19/2023, for the treatment of relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy. Epcoritamab was approved with a boxed warning regarding Cytokine Release Syndrome and Immune Effector Cell-Associated Neurotoxicity Syndrome An FDA review of RSVpreF for the prevention of lower respiratory tract disease caused by RSV in infants, when administered to pregnant mothers, found the vaccine to be efficacious and estimated vaccine efficacy to be 57.1% within 90 days of birth and 51.3% during the first 180 days. RSVpreF was generally safe and had an adverse effect profile similar to placebo (16.2% and 15.2%). It was noted that while there was a numerically higher occurrence of premature delivery, the difference was not significant (5.6% vs 4.7%). The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted 14 to 0 on effectiveness and 10 to 4 on safety for RSVpreF for the prevention of lower respiratory tract disease caused by RSV in infants, when administered to pregnant mothers. Members who voted no for safety were concerned about the higher incidence of premature deliveries. Elevar Therapeutics submitted an NDA for apatinib plus camrelizumab for the treatment of hepatocellular carcinoma. The FDA accepted the BLA for TAK-755 for the treatment of congenital thrombotic thrombocytopenic purpura, which suggests a PDUFA date on 11/16/2023. Mustang Bio announced it would delay initiation of Phase III trials for MB-107 and MB-207 until after a research partner completes a trial evaluating a modified version of the current lentiviral vector. Mustang Bio will initiate the Phase III trials utilizing the safest vector. The FDA granted isaralgagene civaparvovec a Fast Track Designation for the treatment of Fabry disease. Regulatory Update
The FDA approved pegunigalsidase alpha (Elfabrio, Chiesi, Protalix BioTherapeutic), on 5/10/2023, for the treatment of adult patients with Fabry disease. Pegunigalsidasealpha was approved with a boxed waring regarding hypersensitivity reactions, including anaphylaxis. The FDA approved fezolinetant (Veozah,Astellas Pharma), on 5/12/2023, to treat moderate to severe vasomotor symptoms caused by menopause. Astellas priced fezolinetant a t$6,600 per year, which is higher than the $2,000 to $2,500 range recommended byICER. The FDA rejected N-803 plus BacillusCalmette-Guerin (BCG) for the treatment of BCG-unresponsive non-muscle invasive bladder carcinoma due to manufacturing deficiencies. The FDA rejected trastuzumab duocarmazine for the treatment of pretreated HER2-positive unresectable locally advanced or metastatic breast cancer and requested additional unspecified information. An FDA review of delandistrogene moxeparvovec questioned whether microdystrophin protein expressionis a valid surrogate endpoint that can be used for approval, since microdystrophin has not demonstrated a pharmacologic effect on a biomarker in the pathway of the disease. The FDA failed to find a benefit for ambulatory DMD patients in a review of delandistrogene moxeparvovectrials. The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 to recommend approval of delandistrogene moxeparvovec. The FDA designated ampreloxetine an Orphan Drug for the treatment of symptomatic neurogenic orthostatic hypotension in patients with multiple system atrophy. We recently added 16 new drugs to the knowledgebase:
Regulatory Update The FDA approved omidubicel (Omisirge, Gamida Cell), on 4/17,2023, to reduce the time to neutrophil recovery and the incidence of infection. In patients with hematologic malignancies planned for umbilical cord blood transplantation following myeloablative conditioning. Omidubicel was approved with a boxed warning regarding infusion reactions, graft versus host disease, engraftment syndrome, and graft failure. The FDA approved tofersen (QALSODY, Biogen), on 4/25/2023, for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. The FDA approved a fecal microbiota product (Vowst, Seres Therapeutics), on 4/26/2023, for the prevention of recurrent C. difficile infection. The FDA approved GSK’s respiratory syncytial virus vaccine (Arexvy), on 5/3/2023, to prevent lower respiratory tract infection caused by RSV in individuals 60 and older. The FDA rejected concizumab and requested additional information related to monitoring, dosing and the manufacturing process. The FDA granted a Fast Track designation to VE303 for the prevention of recurrent Clostridioides difficile infection. The FDA’s Antimicrobial Drugs Advisory Committee voted 12-0 to recommend approval of the combination of sulbactam and durlobactam for the treatment of hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia caused bysusceptible strains of Acinetobacter. Sulbactam/durlobactam has a PDUFA date of May 29, 2023. The FDA has placed a hold on Sun Pharma’s deuruxolitinib 12 mg due to a report of a pulmonary embolism in a long-term Open Label Extension (OLE) study. The gold does not affect the 8 mg dose, since no thrombotic events have been reported with the lower dose. The European Commission granted marketing authorization for vadadustat (Vafseo, Akebia Therapeutics) for the treatment of anemia in chronic kidney disease (CKD) dialysis patients. Roche obtained zinpentraxin in February 2020, when it bought Promedior. Roche discontinued development of zinpentraxin, in February 2023, as a treatment for idiopathic pulmonary fibrosis after a futility analysis failed to find a benefit compared to placebo. In April 2023, Roche also abandoned development of zinpentraxin to treat myelofibrosis as part of a pipeline review and reprioritization. |
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