This week, new progress has been made in several areas of rare diseasesI haven't heard about medicine in a weekLet's see the detailed report< br / > on June 4, the US Food and Drug Administration (FDA) accepted the biological product license application (BLA) of pharmacoessentia company ropeginterferon α - 2b (p1101) for the treatment of polycythemia vera (PV) without splenomegalyThe drug was approved for marketing by the European Drug Administration (EMA) in 2019< br / > on the same day, tagiri biological obtained the first clinical approval, and tgrx-678, a new generation of BCR ABL inhibitor, was approved in China for the treatment of chronic myeloid leukemia (CML)The drug can significantly prolong the remission time without treatment, and even achieve the effect of long-term discontinuation and prevention of recurrence< br / > at the same time, new news came from the field of B-cell non-Hodgkin's lymphoma treatmentAccording to a 3-phase clinical research data published in the New England Journal of Medicine (NEJM), the 3-year survival rate of children with advanced B-cell non-Hodgkin's lymphoma (B-NHL) can be increased to 95% by adding rituximab on the basis of standard chemotherapy scheme< br / > on June 8, global blood therapeutics (GBT) announced an agreement with the FDA to expand the applicable population of oxbryta (voxelotor) for the treatment of children aged 4 to 11 with SCDPreviously, the drug was approved by the FDA for treatment of adults and adolescents aged 12 and above, and the expansion of the applicable population will probably achieve early intervention of the disease< br / > on the same day, CASI pharmaceuticals submitted a clinical trial application (CTA) for monoclonal antibody therapy cid-103 to MHRA for multiple myeloma (mm) and other hematological malignanciesThis therapy has shown good efficacy and safety advantages in preclinical research< br / > at the EAACI meeting on June 6, positive data came from a number of clinical trials< br / > according to the latest phase 2 clinical trial data released by blueprint medicine, its precise protein kinase inhibitor avapritinib can significantly improve the skin symptoms of patients with systemic mastocytosis (SM)After 24 weeks of treatment, 60% of the patients in the avapritinib treatment group had a remission rate * compared with 0% in the placebo group(* Note: remission is defined as a reduction in the overall symptom score of more than 30%) < br / > in the treatment of hereditary angioedema (HAE), new data from two clinical trials (apex-2 and apex-s) published by biocryst company show that bertralstat (once a day, oral) can continuously reduce the disease attack rate of patients with hereditary angioedema within 48 weeks, and improve the quality of life (QOL) score The two studies also showed good safety and tolerability of bertralstat within 48 weeks < br / > Takeda also published the interim analysis results of three phases of help (long-term prevention of hereditary angioedema) study in the same period, which showed that in different subgroups of patients, lanadelumab (trade name: takhzyro) was well tolerated, which could prevent the onset of hereditary angioedema and continuously reduce the monthly incidence < br / > on November 11, the application for listing of acibant acetate injection developed by shire company of Takeda in China was accepted In 2011, etibant was approved by the US FDA for the treatment of hereditary angioedema acute attack in adults aged 18 and over This is the first time that the original research of etibant has submitted an application for listing in China < br / > on June 6, Sanofi held the fabrizan China listing meeting, and launched the "life praise" fabrizan disease patient assistance project together with China primary health care foundation Fabuzen (AGA β for injection) is the first specific drug for Fabry disease on the market in China, which is used for long-term enzyme replacement therapy of adult patients with Fabry disease (α - galactosidase a deficiency) or children and adolescents aged 8 and over < br / > 7, Alnylam company announced its positive results in the phase 3 study of lumasiran in the treatment of type I primary hyperoxaluria (Ph1) In the study, illuminate-a, urine oxalate levels in patients treated with lumasiran were reduced by an average of 53.5% compared with placebo < br / > on June 8, Boehringer Ingelheim announced that the listing application of its pulmonary fibrosis treatment drug, ofev (nedanib ethylsulfonate soft capsule), was approved by China National Drug Administration (nmpa), becoming the first and only drug currently used for systemic sclerosis related interstitial lung disease (SSC ILD) < br / > on October 10, the European Commission (EC) approved kalydeco (ivacftor), a cystic fibrosis treatment drug from vertex pharmaceuticals, to expand its application to children and adolescents with cystic fibrosis (CF) whose age is 6 months or more, weight is 5 kg or more, and whose cystic fibrosis transmembrane conduction regulator (CFTR) is based on R117H mutation < br / > on June 8, deciphera announced that the results of Invictus, a key three-stage study of ripretinib in the treatment of gastrointestinal stromal tumors (GIST), had been published online in the Lancet Oncology The results showed that riprotinib significantly improved PFS, and had good safety and tolerance < br / > on October 10, the drug evaluation center (CDE) of the State Food and Drug Administration announced that the new drug market application of risdiplam oral solution powder, a SMN2 gene splicing regulator owned by Roche, would be included in the priority review If approved, it will not only become the third global drug for spinal muscular atrophy (SMA), but also the first oral drug for all three types of SMA ▽
