Pipeline News and Updates
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Regulatory Update
The FDA approved tremelimumab (Imjudo, AstraZeneca) in combination with durvalumab, on 10/24/2022, for the treatment of unresectable hepatocellular carcinoma. The FDA approved teclistamab (Tecvayli, Janssen), on 10/25/2022, for the treatment of relapsed or refractory multiple myeloma in patients who previously received four or more prior lines of therapy, including a proteasome inhibitor, immunomodulatory drug and anti-CD38 monoclonal antibody. Teclistamab was approved with a boxed warning for Cytokine Release Syndrome (CRS) and Neurologic Toxicity. The FDA rejected bulevirtide due to concerns regarding the manufacture and delivery of the drug. Daprodustat
The FDA accepted the NDA for quizartinib for the treatment of acute myeloid leukemia (AML) that is FLT3-ITD positive and set a PDUFA date for 4/24/2023. The FDA accepted the BLA for SER-109 for the prevention of recurrent C. difficile infection and set a PDUFA date for 4/26/2023. Regulatory Update
The FDA accepted the resubmitted BLA for BioMarin’s valoctocogene roxaparvovec, for the treatment of severe hemophilia A, and set a PDUFA date for 3/31/2023. The FDA delayed the PDUFA date for Biogen’s tofersen, by three months to 4/25/2023, and requested new information The FDA granted Fast Track status to Nanoscope’s MCO-010 for the treatment of retinitis pigmentosa Pharming submitted an MAA for leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome. CHMP recommended approval of:
Regulatory Update
The UK’s MHRA granted marketing authorization for baricitinib for the treatment of severe alopecia areata. In October 2022, Sanofi signed an agreement with Provention to promote teplizumab in the U.S. Teplizumab has a PDUFA date of 11/17/2022. Published Research Updates A meta-analysis of 26 randomized trials of 1,736 patients with type 1 hepatorenal syndrome (HRS) found that treatment with terlipressin increased HRS reversal compared with placebo (142 reversals per 1,000 patients) and may decrease mortality.
The FDA has updated to the EUA for the COVID-19 monoclonal antibody combination of tixagevimab plus cilgavimab (Evusheld) to indicate that Omicron subvariant BA.4.6 is not susceptible to the antibody combination. Regulatory Update
The FDA approved the combination of sodium phenylbutyrate and taurursodiol (Relyvrio, Amylyx Pharmaceuticals), on 9/29/2022, to slow the functional decline in Amyotrophic Lateral Sclerosis (ALS). Amylyx set WAC for Relyvrio at $158,000 per year. This is slightly less than the cost of intravenous edaravone, but much higher than ICER’s recommended health-benefit price benchmark range of $9,100 to $30,700. The FDA approved futibatinib (Lytgobi, Taiho Oncology), on 9/30/2022, for the treatment of unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 gene fusions or other rearrangements. The FDA accepted the NDA for leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome and assigned a PDUFA date of 3/29/23. BioMarin resubmitted a BLA for valoctocogene roxaparvovec to treat severe hemophilia A with two outcomes from the Phase III GENEr8-1 trial and five years data form a Phase I/II trial. Sarepta submitted a BLA for delandistrogene moxeparvovec for the treatment of ambulant Duchenne muscular dystrophy in September 2022. The EMA accepted the MAA for fezolinetant for the treatment of moderate to severe vasomotor symptoms associated with menopause. |
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July 2023
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