Pipeline News and Updates
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Regulatory Update
The FDA approved lutetium Lu 177 vipivotide tetraxetan (Pluvicto, Novartis), on 3/23/2022, for the treatment of prostate-specific membrane antigen–positive metastatic castration-resistant prostate cancer. Lutetium Lu 177 vipivotide tetraxetan was formerly referred to as 177Lu-PSMA-617. The FDA has also approved gallium Ga 68 gozetotide injection (Locametz, Novartis), a radiolabeling diagnostic agent used to identify PSMA-positive lesions through a positron emission tomography (PET) scan. The FDA followed the ODAC’s recommendation and rejected sintilimab and recommended a multiregional trial that uses a non-inferiority design with an overall survival endpoint before re-submitting the drug. The FDA rejected zandelisib and recommended a Phase III trial to support safety and efficacy. The FDA accepted the re-submitted BLA for teplizumab for the delay of clinical type 1 diabetes in at-risk patients and set a PDUFA date for 8/17/2022. An FDA review of AMX0035 found weak evidence to support efficacy due to loss of patients, the potential for unblinding from adverse effects and only a modest p-value using non-preferred analysis methods. The FDA did not find the open-label, long-term extension data to support a survival benefit. Announced Research Updates Protalix announced results from the Phase III BRIGHT trial, evaluating the impacet of switching Fabry disease patients from agalsidase alfa or beta to pegunigalsidase alfa ImmunoGen announced results from the SORAYA trial, evluating mirvetuximab soravtansine in the treatment of platinum-resistant ovarian cancer. Sangamo announced one-year interim results for four Fabry disease patients treated with a single infusion of the gene therapy, isaralgagene civaparvovec. Pfizer announced results from the ELEVATE UC 12 trial, evaluating etrasimod in the treatment of ulcerative colitis in patients who had failed or were intolerant to at least one conventional biologic, or JAK therapy. UCB announced open-label, long-term extension data from the Phase IIIB, BE RADIANT trial, bimekizumab in the treatment of moderate-to-severe chronic plaque psoriasis. Lilly announced the Phase III, ADvocate 1 & 2 trials, evaluating lebrikizumab in the treatment of moderate-to-severe atopic dermatitis. Published Research Updates The results from the Phase III, NRG-GY004 trial were published describing the effect of olaparib on the treatment ofplatinum-sensitive relapsed ovarian cancer. Long-term results from the explorer4 and explorer5 trials were published evaluating the effect of treatment with concizumab on the annualized bleeding rate in hemophilia A. Regulatory Update
The FDA approved the combination of nivolumab and relatlimab (Opdualag, Bristol-Myers Squibb), on 3/18/2022, for the treatment of unresectable or metastatic melanoma. The FDA approved ganaxolone (Ztalmy, Marinus pharmaceuticals), on 3/18/2022, for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder. ganaxolone is expected to be available in July following scheduling by the DEA. Phantom submitted an NDA to the FDA for the use of vonoprazan to treat all grades of erosive esophagitis. Sodium phenylbutyrate/taurursodiol (AMX0035) is available through an Expanded Access Program (NCT05286372). Announced Research Updates Astellas announced interim results for the Phase III, fezolinetant MOONLIGHT 1 trial. Allena announced interim results from the URIROX-2 trial which lead to the discontinuation of reloxaliase development. Amicus announced 36-month results of treatment with cipaglucosidase alfa/miglustat in patients with Pompe disease. Published Research Updates The results of EMERGE and ENGAGE were published in a single article in the Journal of Prevention of Alzheimer’s Disease. The data presented in the article is the same as has been previously announced. The article did not address limitations identified in the FDA statistical analysis of the trials nor add any insights. One-year results were published for the GENEr8-1 trial, which evaluated a single dose of valoctocogene roxaparvovec in the treatment of severe hemophilia A. Announced Research Updates
Valneva announced Phase III results for their VLA1553 chikungunya vaccine. Nektar and BMS announced results from the PIVOT IO-001 trial evaluating the effect of adding bempegaldesleukin to nivolumab in the treatment of unresectable or metastatic melanoma. Ascendis announced results from the PaTHway trial evaluating the effect of TransCon PTH on the treatment of chronic hypoparathyroidism. Published Research Updates The results of a Phase II Chinese trial evaluating pyrotinib plus capecitabine in the treatment of progressive HER2-positive brain metastases after radiotherapy were published. The results of a Phase III trial evaluating mavrilimumab plus prednisone in the treatment of giant cell arteritis were published. A pharmacoeconomic analysis was published that used data from six Phase II trials and one Phase III trial to estimate cost-effectiveness of using MDMA-assisted therapy (MDMA-AT) to treat severe PTSD. Regulatory Update
The FDA approved ciltacabtagene autoleucel (Carvykti, Johnson & Johnson and Legend Biotech), on 2/28/2022, for the treatment of patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.
The FDA rejected lenacapavir due to concerns of the incompatibility of vials made of borosilicate glass with lenacapavir solution, which could potentially lead to the formation of sub-visible glass particles. Gilead plans to resubmit the NDA with a different vial type. The FDA designated Pfizer’s investigational respiratory syncytial virus (RSV) vaccine, PF-06482077, a Breakthrough Therapy for prevention of RSV-associated lower respiratory tract illness in infants from birth up to six months of age by active immunization of pregnant women. Announced Research Updates Vaccine efficacy for PF-06425090 Clostridium difficile vaccine to prevent Clostridioides difficile in adults 50 years of age and older. AP-013 effect on the WOMAC pain and function scores in patients with severe osteoarthritis of the knee. Safety of fezolinetant on endometrial health in patients with moderate to severe vasomotor symptoms associated with menopause. Published Research Updates Effect of roluperidone on the PANSS-derived Negative Symptoms Factor Score in patients with schizophrenia presenting with moderate to severe negative symptoms. Nirsevimab efficacy in preventing RSV-associated lower respiratory tract infection and hosptializations in healthy late preterm and term infants of 35 weeks or greater gestational age The results form adding toripalimab to paclitaxel plus cisplatin in Chinese patients with advanced esophageal squamous cell carcinoma. The effect of deucravacitinib on achievement of ACR-20 in patients with psoriatic arthritis. Use of camrelizumab, famitinib and nab-paclitaxel in Chinese patients with refractory metastatic immunomodulatory triple-negative breast cancer. The efficacy of gefapixant on the 24-hour coughs per hour average the Phase III COUGH-1 and COUGH-2 trials. |
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