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- Read more about BioMarin receives Orphan Drug Designation from FDA for BMN 270
BioMarin Pharmaceutical Inc. Announced that BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). BioMarin is currently conducting a Phase 1/2 study to evaluate the safety and efficacy of BMN 270 gene therapy in up to 12 patients with severe hemophilia A and will provide a program update in April. BMN 270 is an AAV 5 factor VIII vector, designed to restore factor VIII plasma concentrations, essential for blood clotting in patients with hemophilia A.

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- Read more about Edwards SAPIEN XT Valve receives FDA approval for Pulmonic Procedures
Edwards Lifesciences Corporation announced U.S. Food and Drug Administration approval to expand use of the Edwards SAPIEN XT transcatheter heart valve for pulmonic valve replacement procedures.
- Read more about FDA approves Roche’s Gazyva (obinutuzumab)
Roche announced that the US Food and Drug Administration (FDA) approved Gazyva® (obinutuzumab) plus bendamustine chemotherapy followed by Gazyva alone as a new treatment for people with follicular lymphoma who did not respond to a Rituxan® (rituximab)-containing regimen, or had their follicular lymphoma return after such treatment. Follicular lymphoma is the most common type of indolent (slow-growing) non-Hodgkin lymphoma (NHL) and accounts for approximately one in five cases of NHL.
- Read more about Protein that promotes brain tumour formation identified
Researchers have found that a protein involved in the breakdown of glucose a process known as glycolysis plays an important role in coordinating cellular processes crucial to cancer metabolism and brain tumour formation.
- Read more about New biomarker in early stage Alzheimer's disease found
Researchers have identified a brain inflammation marker in patients at early asymptomatic stages of Alzheimer's disease.This molecule may provide clinicians with a rapidly detectable biomarker for the transition from preclinical Alzheimer's disease to cognitive impairment and progression to full dementia.
- Read more about New kind of stem cell could advance regenerative medicines
Scientists have discovered a new kind of stem cell one that could lead to advances in regenerative medicine as well as offer new ways to study birth defects and other reproductive problems.
- Read more about New surveillance network to track newborns with defects
The World Health Organization has set up a Newborn and Birth Defect Surveillance network, a initiative to keep a track of newborns with defects across the globe.
- Read more about Reconsider decision on vital drugs: CPI-M
The CPI-M on Thursday expressed deep concern that 76 life saving drugs have been taken off the customs duties exemption list and urged the government to review the decision.
- Read more about Novel immunotherapy may beat brain cancer
Researchers, including one of Indian-origin, have shown that a next-generation cell-based immunotherapy may offer new hope in the fight against the most aggressive form of brain cancer called glioma.
- Read more about Abeona Therapeutics Announces FDA Allowance of IND for Systemic AAV Phase 1/2 Clinical Study With ABO-102 Gene Therapy
Abeona Therapeutics, Inc. announced the FDA cleared the Company's Investigational New Drug Application for ABO-102 (AAV- SGSH), a single treatment strategy for Mucopolysaccharidosis Type IIIA (MPS IIIA). The ABO-102 IND application is now active and enables Nationwide Children's Hospital (Columbus, OH) to initiate a Phase 1/2 clinical study designed to assess the safety, tolerability and potential efficacy of ABO-102 in children with MPS IIII A.