1、 Avalglucosidase alfa (nexviazyme)
On August 6, 2021, the US FDA approved Sanofi avalglucosidase alfa (trade name: nexviazyme) to be listed for the treatment of patients aged 1 and over for the treatment of late-onset Pompeii’s disease. Note: Pompeii disease is also known as glycogen accumulation disease type II, acidic- α- Glucosidase deficiency. Previously, FDA granted nexviazyme breakthrough therapy qualification and fast track qualification for Pompeii disease. This approval is based on the positive results obtained from the key phase 3 clinical trial comet. The test data show that nexviazyme can provide patients with the improvement of walking distance and respiratory function indicators.
2、 Welireg (belzutifan)
On August 13, 2021, FDA approved oral hypoxia inducible factor-2 of methadone α (HIF-2 α) The inhibitor welireg (belzutifan) is listed for the treatment of diseases related to von Hippel Lindau syndrome (VHL), including renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastoma or pancreatic neuroendocrine tumor (PNET). These patients do not need immediate surgery. Previously, FDA granted welireg the qualification of breakthrough therapy for this indication.
Welireg is the first HIF-2 approved by FDA α Inhibitor therapy. By inhibiting HIF-2 α， Welireg can reduce HIF-2 related to cell proliferation, angiogenesis and tumor growth α Transcription and expression of target genes.
This approval was based on data from the open label phase 2 study-004 study (nct03401788), which showed that welireg treatment showed lasting remission in patients with VHL related RCC (n = 61), VHL related CNS hemangioblastoma (n = 24) and VHL related PNET (n = 12).
3、 Difelikefalin (korsuva)
On August 23, 2021, Cara therapeutics and VIFor Pharma jointly announced that FDA had approved difelikefalin (korsuva) to be listed for the treatment of moderate and severe pruritus associated with hemodialysis (hd) adult chronic kidney disease (CKD AP).
This is the first and only therapy approved by the FDA for the treatment of chronic kidney disease-related pruritus. Previously, the FDA granted difelikefalin the qualification of breakthrough therapy for this indication.
This approval was based on positive data from two key phase 3 clinical trials, including the kalm-1 trial in the United States and the global kalm-2 trial, as well as supporting data from an additional 32 clinical studies. In the two key phase 3 clinical trials, korsuva reached the primary end point of the trial, and more patients treated with korsuva were evaluating wi-nrs of pruritus compared with placebo The score improved by more than 3 points.
4、 Transcon human growth hormone (skytrofa)
On August 25, 2021, ascendis pharmaceutical transcon human growth hormone (lonapegsomatropin) was approved by FDA for the treatment of growth hormone deficiency (pghd) in children. It is the first product approved by FDA that can continuously release and deliver growth stimulating hormone within one week through one injection.
The new automatic syringe and cartridge of transcon human growth hormone are also approved this time. The device can store the drugs at room temperature for up to six months after they are first removed from the refrigerator. Compared with daily injection, patients injected every week can reduce the number of injection days per year by 86%.