A $3.5 million gene therapy for Hemophilia B

Etranacogene dezaparvovec, marketed as Hemgenix, is the first gene therapy for adults with Hemophilia B to be approved. It is an adeno-associated virus vector-based gene therapy and is indicated for hemophilia B patients who have suffered potentially fatal hemorrhage (current or past) or frequent spontaneous bleeding episodes or are on Factor IX prophylaxis therapy. It is a one-time gene therapy product to be administered as a single dose by IV infusion. Priced at $3.5 million, it is the costliest treatment up till now…(Source: Nov. 22, 2022).

Risk of severe hypocalcemia with denosumab in patients on dialysis

In a safety alert, the US FDA has cautioned about the risk of severe hypocalcemia with denosumab in patients with advanced kidney disease undergoing dialysis. A mortality risk has also been highlighted. The FDA has asked patients to continue taking the treatment and not discontinue it without the advice of the treating doctor. It has also advised clinicians to regularly monitor blood calcium levels in these patients along with calcium and vitamin D supplementation. Denosumab, a human monoclonal antibody, is used for the treatment of osteoporosis…(Source: Nov. 22, 2022).

Mirvetuximab soravtansine gets accelerated approval for folate receptor alpha–positive ovarian cancer

Earlier this month, mirvetuximab soravtansine-gynx was accorded accelerated approval for the treatment of adult patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer whose tumors test positive for folate receptor alpha (FRα) expression. These patients had already undergone 1-3 systemic treatment cycles. The FDA also approved “a companion diagnostic device” Ventana FOLR1 (FOLR-2.1) RxDx Assay to identify patients suitable for this therapy… (Source: US FDA, Nov. 14, 2022).

Piziotinib rejected for non-small cell lung cancer 

Poziotinib, a drug developed by Spectrum Pharmaceuticals has failed to get FDA nod because of insufficient clinical data. The drug was intended for the treatment of patients with previously treated locally advanced or metastatic non-small cell lung cancer (NSCLC) who have a rare HER2 exon 20 insertion mutations. Poziotinib is an oral pan-HER tyrosine kinase inhibitor. Earlier too in September the drug had been voted 9-4 against approval…(Source: Medscape, Nov. 25, 2022).