Tags : Aged

Vertex’s Kaftrio + Ivacaftor Receive the EC’s Approval to Treat

Shots: The EC has granted MAA to Kaftrio (ivacaftor/tezacaftor/lumacaftor) + ivacaftor (150mg) to treat people with CF aged ≥12yrs. with one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the CFTR gene The MAA is based on two P-III studies i.e. a 24wks. study in 403 people with one […]Read More

Merck & Co’s Dificid (fidaxomicin) Receives FDA’s Approval to Treat

Shots: The US FDA has approved NDA for Dificid oral suspension and sNDA for Dificid tablets for the treatment of Clostridioides difficile-associated diarrhea (CDAD) in patients aged ≥6mos. The approval is based on P-III SUNSHINE study assessing Dificid (bid) vs vancomycin (q4d) in 148 patients in a ratio (2:1) aged <18 yrs. with confirmed CDI […]Read More

Amgen’s Nplate (Romiplostim) Receives FDA’s Approval for Immune Thrombocytopenia Aged

Shots: The approval is based on P-III & P-I/II study results assessing Nplate vs PBO in pediatric patients with Immune Thrombocytopenia (ITP) for at least six months who had no response to corticosteroids, immunoglobulins or splenectomy The  P-III & P-I/II study demonstrated increase in overall platelet response (71% vs 20%) with durable platelet response (52% vs 10%) Nplate is a thrombopoietin […]Read More

Vertex‘s Orkambi (lumacaftor/ivacaftor) Receives Health Canada Approval for Cystic Fibrosis

Shots: The approval is based on P-III study results assessing Orkambi in 60 patients with CF, who have two copies of the F508del CFTR mutation aged 6 to 11 years for 24 wks. The P-III study demonstrated in reduction of sweat chloride by 31.7 mmol/L and was well-tolerated, Presented at European Cystic Fibrosis Society Conference in June 2018 Orkambi is a combination […]Read More