Siremadlin

AstraZeneca is currently analysing Calquence to treat various multiple B-cell blood cancers. Credit: Jonathan Weiss/Shutterstock.com. The Chinese National Medical Products Administration (NMPA) has granted approval for AstraZeneca ’s Calquence (acalabrutinib) to treat chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL) in adults. A selective inhibitor of Bruton’s tyrosine kinase (BTK), Calquence is indicated for use in CLL or SLL patients who have received a minimum of one treatment previously. Recommended Reports Reports LOA and PTSR Model - Siremadlin Succinate in Well Differentiated Liposarcoma GlobalData Reports LOA and PTSR Model - Ribociclib Succinate in Well Differentiated Liposarcoma GlobalData View all Companies Intelligence AstraZeneca Plc Ascend GmbH View all The approval was based on positive data from two clinical trials; namely, the Phase III ASCEND trial of Calquence in relapsed or refractory (R/R) CLL patients and a Phase I/II trial in R/R CLL patients in China. According to the data from the Phase I/II trial, treatment with Calquence offered an overall response rate of 83.3% in study subjects. ASCEND findings showed that 88% of trial subjects were alive and free from disease progression after 12 months following treatment with Calquence. AstraZeneca oncology business unit executive vice-president Dave Fredrickson said: “The approval is another step towards our goal of making Calquence available to as many patients as possible and offering physicians a treatment option with a well-established efficacy and tolerability profile. “Patients with chronic lymphocytic leukaemia are often older and dealing with significant comorbidities, and tolerability is a critical factor in their treatment.” Calquence is currently available for treating CLL and SLL in the US and Japan while it is indicated for CLL in the R/R and treatment-naïve patients in the EU and other countries globally. Apart from CLL, the product is also being analysed by the company to treat multiple B-cell blood cancers; namely, mantle cell lymphoma (MCL), diffuse large B-cell lymphoma, Waldenström’s macroglobulinaemia and marginal zone lymphoma, among others. In March 2023, the Chinese regulator granted conditional approval for Calquence for the treatment of MCL in adults who have received a minimum of one earlier therapy.

Phalguni Deswal @Phalguni_GD Veopoz’s approval was based on the data from the open-label Phase II/III trial (NCT04209634) consisting of ten participants. Image Credit: lev radin / Shutterstock. Regeneron Pharmaceuticals has received US Food and Drug Administration (FDA) approvals for two of its drugs, namely Veopoz (pozelimab-bbfg) and high-dose Eylea (aflibercept) in the last few days. Veopoz is the first drug which has been approved by the FDA for CD55 deficiency with hyperactivation of complement, angiopathic thrombosis, and protein-losing enteropathy or CHAPLE disease in patients aged one year and older. Recommended Reports Reports LOA and PTSR Model - Siremadlin Succinate in Myelodysplastic Syndrome GlobalData Reports LOA and PTSR Model - Docetaxel Albumin Bound in Fallopian Tube Cancer GlobalData View all Companies Intelligence Bayer AG Regeneron Pharmaceuticals Inc Alnylam Pharmaceuticals Inc Regeneron GmbH View all CHAPLE is a genetic immune disease caused by mutations in the CD55 gene causing overactivation of the complement system. This results in impaired inflammatory response and causes symptoms such as abdominal pain, recurrent infections, oedema, and diarrhoea. Veopoz , a monoclonal antibody, targets and inhibits the protein involved in the activation of the complement system, the C5 complement factor. Additionally, Eylea HD, which is being codeveloped by Regeneron and Bayer , was granted approval to treat ophthalmologic disorders such as wet age-related macular degeneration (wAMD), diabetic retinopathy (DR) and diabetic macular oedema (DME). Veopoz’s approval was based on the data from the open-label Phase II/III trial (NCT04209634) consisting of 10 participants, who all achieved the primary outcome of normalised serum albumin at 12 weeks, with the patients maintaining these levels over 72 weeks of treatment. At 48 weeks, only one patient required an albumin transfusion, compared to the period of 48 weeks before starting treatment, when five patients required a total of 60 transfusions. Additionally, two patients were hospitalised for seven days in 48 weeks after starting treatment, compared to nine patients requiring hospitalisation for a cumulative 268 days in the period before starting treatment. Veopoz is administered through a 30mg/kg intravenous loading dose, followed by weekly weight-based subcutaneous doses. The most commonly observed adverse events were upper respiratory tract infection, fracture, urticaria (itchy rash), and hair loss. Approved therapies for rare diseases have increased in numbers in recent years, with GlobalData forecasting at least 35 US FDA approval in the rare disease areas in 2023. GlobalData is the parent company of Pharmaceutical Technology . Veopoz is also being investigated in other complement system-mediated diseases, including paroxysmal nocturnal haemoglobinuria (PNH) and myasthenia gravis. The combination therapy of Veopoz and Alnylam Pharmaceuticals’ cemdisiran, a small interfering RNAi C5 inhibitor , is being investigated in an open-label Phase II trial (NCT04888507).