| |JANUARY 20248 US FDA NODS MERCK'S THERAPY TO TREAT PATIENTS WITH CERVICAL CANCERDRUG FARM BAGS US FDA RPD TO TREAT ROSAH SYNDROMEThe US Food and Drug Administration (FDA) has approved Merck's anti-PD-1 therapy, Keytruda, in conjunction with chemotherapy radiation therapy (CRT) for the treatment of patients with FIGO (International Federation of Gynecology and Obstetrics) 2014 Stage III-IVA cervical cancer. Merck is referred to as MSD outside of the US and Canada.The approval is based on data from the phase 3 KEYNOTE-A18 trial, in which Keytruda plus CRT demonstrated an improvement in progression-free survival (PFS), reducing the risk of disease progression or death by 41 percent (HR=0.59 [95 percent CI, 0.43-0.82]) compared to placebo plus CRT in patients with FIGO 2014 Stage III-IVA disease. Median PFS was not reached in either group. This approval marks the third indication for Keytruda in cervical cancer and the 39th indication for Keytruda in the US, as per pharmabiz."Today's approval of Keytruda plus chemoradiotherapy is welcome news and gives patients with newly diagnosed FIGO 2014 Stage III-IVA cervical cancer, for the first time ever, the option of an anti-PD-1-based regimen to treat their cancer," said Dr. Bradley Monk, oncologist and professor of obstetrics and gynaecology at University of Arizona's College of Medicine and Creighton University School of Medicine. "This Keytruda-based regimen offers a new treatment option for these patients, so today's approval has important implications for the way we treat them moving forward."Immune-mediated adverse reactions, which can be severe or lethal, can occur in any organ or tissue and affect several physiological systems at the same time. Keytruda medication can cause immune-mediated adverse responses such as pneumonitis, colitis, hepatitis, endocrinopathies, nephritis, dermatologic reactions, solid organ transplant rejection, and problems of allogeneic hematopoietic stem cell transplantation. The important immune-mediated adverse reactions listed here may not contain all potentially severe and fatal immune-mediated adverse events. PODrug Farm, a clinical-stage biopharmaceutical company announced that the US Food and Drug Administration (FDA) has granted Rare Paediatric Disease (RPD) Designation for the company's alpha-kinase 1 (ALPK1) inhibitor, DF-003, to treat patients with ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) syndrome. DF-003 is currently being evaluated in a phase 1 trial (NCT05997641) to assess safety and pharmacokinetics in normal healthy volunteers."Paediatric patients living with ROSAH syndrome face a significant unmet need with limited options to treat vision loss," said Jeysen Yogaratnam, chief medical officer, Drug Farm. "Obtaining Rare Paediatric Disease Designation recognizes the serious and debilitating complications of this rare disease and upholds our goal to provide DF-003 as the first targeted drug for potential treatment in patients afflicted with ROSAH syndrome."Rare Paediatric Disease (RPD) Designation is granted by the FDA for serious or life-threatening diseases which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If a New Drug Application (NDA) for DF-003 to treat ROSAH Syndrome is approved by the FDA, Drug Farm may be eligible to receive a Priority Review Voucher (PRV) that can be redeemed to receive a priority review for any subsequent marketing application or may be sold or transferred. The FDA has implemented this programme to encourage drug development for treatment of rare paediatric diseases.ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) Syndrome is a rare, autosomal dominant autoinflammatory disease named according to the characteristic symptoms exhibited by affected patients. The disease is caused by a genetic gain-of-function mutation in ALPK1. PO
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